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and blood progenitors umbilical cord-derived expanded with (1R, 4R)-N1-(2-benzyl-7-(2-methyl-2H-tetrazol-5-yl)-9H-pyrimido[4,5-b]indol-4-yl)cyclohexane-1,4-diamine dihydrobromide dihydrate", "date_of_designation_or_refusal": "20/04/2020", "intended_use": "Treatment in haematopoietic stem cell transplantation", "eu_designation_number": "EU/3/20/2271", "status": "Positive", "first_published_date": "12/08/2020", "last_updated_date": "20/06/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2271" }, { "medicine_name": "Vyjuvek", "related_ema_product_number": "EMEA/H/C/006330", "active_substance": "genetically modified replication-incompetent herpes simplex virus-1 expressing collagen VII (beremagene geperpavec)", "date_of_designation_or_refusal": "16/04/2018", "intended_use": "Treatment of epidermolysis bullosa", "eu_designation_number": "EU/3/18/2012", "status": "Positive", "first_published_date": "17/05/2018", "last_updated_date": "10/06/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2012" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "adeno-associated virus serotype rh79 containing the human OTC gene, adeno-associated virus serotype rh79 encoding a meganuclease for targeted editing of the human PCSK9 gene", "date_of_designation_or_refusal": "09/12/2022", "intended_use": "Treatment of ornithine transcarbamylase deficiency", "eu_designation_number": "EU/3/22/2736", "status": "Positive", "first_published_date": "13/03/2023", "last_updated_date": "10/06/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2736" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Humanised IGg1 monoclonal antibody targeting human transferrin receptor conjugated to human iduronate-2-sulfatase", "date_of_designation_or_refusal": "26/02/2019", "intended_use": "Treatment of mucopolysaccharidosis type II (Hunter's syndrome)", "eu_designation_number": "EU/3/19/2140", "status": "Positive", "first_published_date": "09/04/2019", "last_updated_date": "10/06/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2140" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "synthetic oligonucleotide selectively targeting UBE3A antisense RNA transcripts", "date_of_designation_or_refusal": "09/12/2020", "intended_use": "Treatment of Angelman syndrome", "eu_designation_number": "EU/3/20/2379", "status": "Positive", "first_published_date": "27/05/2021", "last_updated_date": "10/06/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2379" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "human IgG1 monoclonal antibody against sortilin", "date_of_designation_or_refusal": "15/10/2021", "intended_use": "Treatment of frontotemporal dementia", "eu_designation_number": "EU/3/21/2498", "status": "Withdrawn", "first_published_date": "20/01/2022", "last_updated_date": "10/06/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2498" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Entospletinib", "date_of_designation_or_refusal": "16/10/2017", "intended_use": "Treatment of acute myeloid leukaemia", "eu_designation_number": "EU/3/17/1922", "status": "Withdrawn", "first_published_date": "08/01/2018", "last_updated_date": "10/06/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1922" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "1-(4-(6-chloropyridazin-3-yl)piperazin-1-yl)-2-(4-cyclopropyl-3-fluorophenyl)ethan-1-one", "date_of_designation_or_refusal": "19/07/2021", "intended_use": "Treatment of propionic acidaemia", "eu_designation_number": "EU/3/21/2462", "status": "Withdrawn", "first_published_date": "12/11/2021", "last_updated_date": "10/06/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2462" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "1-(4-(6-chloropyridazin-3-yl)piperazin-1-yl)-2-(4-cyclopropyl-3-fluorophenyl)ethan-1-one", "date_of_designation_or_refusal": "19/07/2021", "intended_use": "Treatment of pantothenate kinase-associated neurodegeneration", "eu_designation_number": "EU/3/21/2461", "status": "Withdrawn", "first_published_date": "12/11/2021", "last_updated_date": "10/06/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2461" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "nerandomilast", "date_of_designation_or_refusal": "28/06/2024", "intended_use": "Treatment of idiopathic pulmonary fibrosis", "eu_designation_number": "EU/3/24/2931", "status": "Withdrawn", "first_published_date": "22/08/2024", "last_updated_date": "10/06/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2931" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "S-[5-(omega-methoxypoly(oxyethylene)-2-oxopentyl)]-L-cysteinylglycyl-L-serinylglycylgylcyl-L-isoleucyl-L-lysyl-L-glutamyl-L-phenylalanyl-L-leucyl-L-glutaminyl-L-arginyl-L-phenylalanyl-L-isoleucyl-L-histyl-L-isoleucyl-L-valyl-L-glutaminyl-L-serinyl-L-isoleucyl-L-isoleucyl-L-asparaginyl-L-threonyl-L-serinamide, acetate salt", "date_of_designation_or_refusal": "13/04/2021", "intended_use": "Treatment of cutaneous T-cell lymphoma", "eu_designation_number": "EU/3/21/2425", "status": "Withdrawn", "first_published_date": "21/01/2022", "last_updated_date": "10/06/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2425" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "adeno-associated viral vector serotype 3B encoding human multidrug resistance protein 3A", "date_of_designation_or_refusal": "22/04/2020", "intended_use": "Treatment of progressive familial intrahepatic cholestasis", "eu_designation_number": "EU/3/20/2267", "status": "Withdrawn", "first_published_date": "11/08/2020", "last_updated_date": "10/06/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2267" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "nipocalimab", "date_of_designation_or_refusal": "", "intended_use": "Prevention of fetal and neonatal alloimmune thrombocytopenia", "eu_designation_number": "EU/3/25/3045", "status": "Positive", "first_published_date": "10/06/2025", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-25-3045" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "sirolimus", "date_of_designation_or_refusal": "16/04/2025", "intended_use": "Treatment of familial adenomatous polyposis (FAP)", "eu_designation_number": "EU/3/25/3051", "status": "Positive", "first_published_date": "10/06/2025", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-25-3051" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Parahydroxybenzoic acid", "date_of_designation_or_refusal": "16/04/2025", "intended_use": "Treatment of primary ubiquinone deficiency", "eu_designation_number": "EU/3/25/3052", "status": "Positive", "first_published_date": "10/06/2025", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-25-3052" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Adeno-associated viral vector serotype LK03 containing the human CFI gene", "date_of_designation_or_refusal": "16/04/2025", "intended_use": "Treatment of primary IgA nephropathy", "eu_designation_number": "EU/3/25/3048", "status": "Positive", "first_published_date": "10/06/2025", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-25-3048" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Fragment antibody targeting human TfR1 conjugated to phosphorodiamidate morpholino oligomer", "date_of_designation_or_refusal": "16/04/2025", "intended_use": "Treatment of Duchenne muscular dystrophy", "eu_designation_number": "EU/3/25/3047", "status": "Positive", "first_published_date": "10/06/2025", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-25-3047" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "defibrotide", "date_of_designation_or_refusal": "29/07/2004", "intended_use": "Prevention of hepatic veno-occlusive disease", "eu_designation_number": "EU/3/04/211", "status": "Withdrawn", "first_published_date": "12/12/2005", "last_updated_date": "05/06/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-04-211" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "defibrotide", "date_of_designation_or_refusal": "13/11/2013", "intended_use": "Prevention of graft-versus-host disease", "eu_designation_number": "EU/3/13/1201", "status": "Withdrawn", "first_published_date": "02/12/2013", "last_updated_date": "05/06/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1201" }, { "medicine_name": "Ztalmy", "related_ema_product_number": "EMEA/H/C/005825", "active_substance": "ganaxolone", "date_of_designation_or_refusal": "13/11/2019", "intended_use": "Treatment of CDKL5 deficiency disorder", "eu_designation_number": "EU/3/19/2224", "status": "Positive", "first_published_date": "13/02/2020", "last_updated_date": "05/06/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2224" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "single guide RNA targeting the human TTR gene, Ziclumeran", "date_of_designation_or_refusal": "26/03/2021", "intended_use": "Treatment of ATTR amyloidosis", "eu_designation_number": "EU/3/21/2419", "status": "Positive", "first_published_date": "19/01/2022", "last_updated_date": "05/06/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2419" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "ganaxolone", "date_of_designation_or_refusal": "15/10/2021", "intended_use": "Treatment of tuberous sclerosis", "eu_designation_number": "EU/3/21/2503", "status": "Positive", "first_published_date": "20/01/2022", "last_updated_date": "05/06/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2503" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "resminostat", "date_of_designation_or_refusal": "09/12/2011", "intended_use": "Treatment of Hodgkin's lymphoma", "eu_designation_number": "EU/3/11/930", "status": "Withdrawn", "first_published_date": "20/12/2011", "last_updated_date": "05/06/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-930" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "recombinant adeno-associated viral vector serotype 5 encoding Staphylococcus aureus Cas9 endonuclease and two guide RNAs complementary to two regions of intron 26 of the CEP290 gene", "date_of_designation_or_refusal": "16/10/2017", "intended_use": "Treatment of Leber's congenital amaurosis", "eu_designation_number": "EU/3/17/1928", "status": "Withdrawn", "first_published_date": "08/01/2018", "last_updated_date": "05/06/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1928" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "resminostat", "date_of_designation_or_refusal": "27/09/2011", "intended_use": "Treatment of hepatocellular carcinoma", "eu_designation_number": "EU/3/11/913", "status": "Withdrawn", "first_published_date": "17/10/2011", "last_updated_date": "05/06/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-913" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "human decorin fused to the truncated homing peptide CRK", "date_of_designation_or_refusal": "18/07/2022", "intended_use": "Treatment of epidermolysis bullosa", "eu_designation_number": "EU/3/22/2664", "status": "Positive", "first_published_date": "23/01/2023", "last_updated_date": "05/06/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2664" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "posnafusp alfa", "date_of_designation_or_refusal": "14/01/2022", "intended_use": "Treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome)", "eu_designation_number": "EU/3/21/2560", "status": "Positive", "first_published_date": "09/06/2022", "last_updated_date": "05/06/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2560" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "autologous T lymphocyte-enriched population of cells transduced with a lentiviral vector encoding a chimeric antigen receptor targeting human claudin 18.2 antigen with CD28 and CD3-zeta intracellular signalling domains", "date_of_designation_or_refusal": "19/02/2021", "intended_use": "Treatment of gastric cancer", "eu_designation_number": "EU/3/21/2405", "status": "Positive", "first_published_date": "23/02/2022", "last_updated_date": "05/06/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2405" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "small-interfering RNA against soluble fms-like tyrosine kinase-1 e15a mRNA, small-interfering RNA against soluble fms-like tyrosine kinase-1 i13 mRNA", "date_of_designation_or_refusal": "16/04/2025", "intended_use": "Treatment of pre-eclampsia", "eu_designation_number": "EU/3/25/3050", "status": "Positive", "first_published_date": "05/06/2025", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-25-3050" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "humanised IgG1 monoclonal antibody against muscle specific kinase", "date_of_designation_or_refusal": "16/04/2025", "intended_use": "Treatment of spinal muscular atrophy", "eu_designation_number": "EU/3/25/3049", "status": "Positive", "first_published_date": "05/06/2025", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-25-3049" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "vamikibart", "date_of_designation_or_refusal": "16/04/2025", "intended_use": "Treatment of non-infectious uveitis", "eu_designation_number": "EU/3/25/3046", "status": "Positive", "first_published_date": "05/06/2025", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-25-3046" }, { "medicine_name": "Ziihera", "related_ema_product_number": "EMEA/H/C/006380", "active_substance": "zanidatamab", "date_of_designation_or_refusal": "19/07/2021", "intended_use": "Treatment of biliary tract cancer", "eu_designation_number": "EU/3/21/2458", "status": "Positive", "first_published_date": "12/11/2021", "last_updated_date": "25/04/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2458" }, { "medicine_name": "Sephience", "related_ema_product_number": "EMEA/H/C/006331", "active_substance": "(S)-2-amino-6-(2-hydroxypropanoyl)-7,8-dihydropteridin-4(3H)-one", "date_of_designation_or_refusal": "20/05/2021", "intended_use": "Treatment of hyperphenylalaninemia", "eu_designation_number": "EU/3/21/2435", "status": "Positive", "first_published_date": "18/03/2022", "last_updated_date": "25/04/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2435" }, { "medicine_name": "Alyftrek", "related_ema_product_number": "EMEA/H/C/006382", "active_substance": "(14S)-8-[3-(2-{dispiro[2.0.24.13]heptan-7-yl}ethoxy)-1H-pyrazol-1-yl]-12,12-dimethyl-2lamba6-thia-3,9,11,18,23-penta-azatetracyclo[17.3.1.111,14.05,10]tetracosa-1(22),5,7,9,19(23),20-hexaene-2,2,4-trione calcium salt hydrate, deutivacaftor, tezacaftor", "date_of_designation_or_refusal": "12/11/2021", "intended_use": "Treatment of cystic fibrosis", "eu_designation_number": "EU/3/21/2527", "status": "Positive", "first_published_date": "07/06/2022", "last_updated_date": "25/04/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2527" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "expanded human allogeneic mesenchymal adult stem cells extracted from adipose tissue (darvadstrocel)", "date_of_designation_or_refusal": "08/10/2009", "intended_use": "Treatment of anal fistula", "eu_designation_number": "EU/3/09/667", "status": "Withdrawn", "first_published_date": "20/10/2009", "last_updated_date": 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"related_ema_product_number": "", "active_substance": "autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19", "date_of_designation_or_refusal": "20/02/2025", "intended_use": "Treatment of myasthenia gravis", "eu_designation_number": "EU/3/24/3011", "status": "Positive", "first_published_date": "13/01/2025", "last_updated_date": "12/03/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-3011" }, { "medicine_name": "Lyvdelzi (previously Seladelpar Gilead)", "related_ema_product_number": "EMEA/H/C/004692", "active_substance": "seladelpar", "date_of_designation_or_refusal": "20/02/2025", "intended_use": "Treatment of primary biliary cholangitis", "eu_designation_number": "EU/3/17/1930", "status": "Positive", "first_published_date": "10/01/2018", "last_updated_date": "12/03/2025", "orphan_designation_url": 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"active_substance": "human anti-promyostatin monoclonal antibody (apitegromab)", "date_of_designation_or_refusal": "14/12/2018", "intended_use": "Treatment of spinal muscular atrophy", "eu_designation_number": "EU/3/18/2115", "status": "Positive", "first_published_date": "07/03/2019", "last_updated_date": "24/02/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2115" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "rilzabrutinib", "date_of_designation_or_refusal": "16/01/2025", "intended_use": "Treatment of autoimmune haemolytic anaemia", "eu_designation_number": "EU/3/24/3019", "status": "Positive", "first_published_date": "20/02/2025", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-3019" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "clofutriben", "date_of_designation_or_refusal": 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"related_ema_product_number": "", "active_substance": "zopapogene imadenovec", "date_of_designation_or_refusal": "12/01/2024", "intended_use": "Treatment of recurrent respiratory papillomatosis", "eu_designation_number": "EU/3/23/2887", "status": "Positive", "first_published_date": "11/04/2024", "last_updated_date": "20/02/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2887" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "N-(2-Methoxyethyl)-6-methyl-N-[(3-methyl-2-thienyl)methyl]-2-oxo-1,2-dihydropyridine-4-carboxamide", "date_of_designation_or_refusal": "16/01/2025", "intended_use": "Treatment of glycogen storage disease type IV", "eu_designation_number": "EU/3/24/3023", "status": "Positive", "first_published_date": "20/02/2025", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-3023" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "volixibat potassium", "date_of_designation_or_refusal": "16/01/2025", "intended_use": "Treatment of primary biliary cholangitis", "eu_designation_number": "EU/3/24/3017", "status": "Positive", "first_published_date": "20/02/2025", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-3017" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "allogenic umbilical cord-derived osteoblast cells", "date_of_designation_or_refusal": "16/01/2025", "intended_use": "Treatment of non-traumatic osteonecrosis", "eu_designation_number": "EU/3/24/3022", "status": "Positive", "first_published_date": "20/02/2025", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-3022" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Elraglusib", 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"orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-3012" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "mitapivat sulfate", "date_of_designation_or_refusal": "13/12/2024", "intended_use": "Treatment of sickle cell disease", "eu_designation_number": "EU/3/24/2999", "status": "Positive", "first_published_date": "15/01/2025", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-2999" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "4-{[(2R,3S,4R,5S)-4-(4-chloro-2-fluoro-phenyl)-3-(3-chloro-2-fluoro-phenyl)-4-cyano-5-(2,2-dimethyl-propyl)-pyrrolidine-2-carbonyl]-amino}-3-methoxy-benzoic acid ", "date_of_designation_or_refusal": "22/08/2014", "intended_use": "Treatment of acute myeloid leukaemia", "eu_designation_number": "EU/3/14/1328", "status": "Withdrawn", "first_published_date": "02/10/2014", 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"orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2081" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "", "date_of_designation_or_refusal": "15/02/2023", "intended_use": "Treatment of Huntington's disease", "eu_designation_number": "EU/3/23/2754", "status": "Withdrawn", "first_published_date": "24/01/2024", "last_updated_date": "15/01/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2754" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "isotretinoin", "date_of_designation_or_refusal": "11/10/2022", "intended_use": "Treatment of autosomal recessive congenital ichthyosis", "eu_designation_number": "EU/3/22/2701", "status": "Withdrawn", "first_published_date": "24/01/2023", "last_updated_date": "15/01/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2701" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "isotretinoin", "date_of_designation_or_refusal": "15/02/2023", "intended_use": "Treatment of recessive X-linked ichthyosis", "eu_designation_number": "EU/3/23/2760", "status": "Withdrawn", "first_published_date": "24/01/2024", "last_updated_date": "15/01/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-23-2760" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "omigapil maleate", "date_of_designation_or_refusal": "08/05/2008", "intended_use": "Treatment of congenital muscular dystrophy with merosin (laminin alpha 2) deficiency", "eu_designation_number": "EU/3/08/544", "status": "Withdrawn", "first_published_date": "29/07/2008", "last_updated_date": "15/01/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-544" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "omigapil maleate", "date_of_designation_or_refusal": "08/05/2008", "intended_use": "Treatment of congenital muscular dystrophy with collagen VI deficiency", "eu_designation_number": "EU/3/08/540", "status": "Withdrawn", "first_published_date": "29/07/2008", "last_updated_date": "15/01/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-540" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "propagermanium", "date_of_designation_or_refusal": "19/11/2018", "intended_use": "Treatment of focal segmental glomerulosclerosis", "eu_designation_number": "EU/3/18/2100", "status": "Withdrawn", "first_published_date": "19/02/2019", "last_updated_date": "15/01/2025", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2100" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "2'-O-4'-C-(S)-ethyl-P-thioadenylyl-(3'-O->5'-O)-2'-O-4'-C-(S)-ethyl-P-thioguanylyl-(3'-O->5'-O)-2'-O-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-fluoro-P-thioadenylyl-(3'-O->5'-O)-2'-fluoro-P-thiocytidylyl-(3'-O->5'-O)-2'-fluoro-P-thiouridylyl-(3'-O->5'-O)-2'-O-methyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-4'-C-(S)-ethyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-4'-C-(S)-ethyl-adenosine", "date_of_designation_or_refusal": "13/12/2024", "intended_use": "Treatment of autosomal dominant polycystic kidney disease", "eu_designation_number": "EU/3/24/3009", "status": "Positive", "first_published_date": "15/01/2025", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-3009" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "monepantel", "date_of_designation_or_refusal": "13/12/2024", "intended_use": "Treatment of amyotrophic lateral sclerosis", "eu_designation_number": "EU/3/24/3004", "status": "Positive", "first_published_date": "15/01/2025", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-3004" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "allogeneic cardiosphere-derived cells", "date_of_designation_or_refusal": "13/12/2024", "intended_use": "Treatment of Duchenne muscular dystrophy", "eu_designation_number": "EU/3/24/3003", "status": "Positive", "first_published_date": "15/01/2025", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-24-3003" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "diazoxide choline", "date_of_designation_or_refusal": "13/12/2024", "intended_use": "Treatment of glycogen storage disease type I", "eu_designation_number": "EU/3/24/3008", "status": "Positive", "first_published_date": "15/01/2025", "last_updated_date": "", "orphan_designation_url": 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"first_published_date": "24/06/2010", "last_updated_date": "13/09/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-732" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "mixture of seven synthetic fragments consisting of p21 RAS peptides", "date_of_designation_or_refusal": "05/08/2011", "intended_use": "Treatment of pancreatic cancer", "eu_designation_number": "EU/3/11/885", "status": "Positive", "first_published_date": "16/08/2011", "last_updated_date": "13/09/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-885" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Pomalidomide", "date_of_designation_or_refusal": "08/10/2009", "intended_use": "Treatment of multiple myeloma", "eu_designation_number": "EU/3/09/672", "status": "Expired", "first_published_date": "20/10/2009", "last_updated_date": "13/09/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-672" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "enzastaurin hydrochloride", "date_of_designation_or_refusal": "20/03/2007", "intended_use": "Treatment of diffuse large B-cell lymphoma", "eu_designation_number": "EU/3/07/442", "status": "Withdrawn", "first_published_date": "10/07/2008", "last_updated_date": "12/09/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-442" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "risdiplam", "date_of_designation_or_refusal": "26/02/2019", "intended_use": "Treatment of spinal muscular atrophy", "eu_designation_number": "EU/3/19/2145", "status": "Withdrawn", "first_published_date": "09/04/2019", "last_updated_date": "12/09/2023", "orphan_designation_url": 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"eu_designation_number": "EU/3/17/1955", "status": "Withdrawn", "first_published_date": "12/03/2018", "last_updated_date": "12/09/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1955" }, { "medicine_name": "Fanskya", "related_ema_product_number": "EMEA/H/C/005537", "active_substance": "Lentiviral vector carrying the Fanconi anaemia-A (FANCA) gene", "date_of_designation_or_refusal": "17/12/2010", "intended_use": "Treatment of Fanconi anaemia type A", "eu_designation_number": "EU/3/10/822", "status": "Positive", "first_published_date": "20/01/2011", "last_updated_date": "11/09/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-822" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "2',3',5'-Tri-O-acetyluridine", "date_of_designation_or_refusal": "15/05/2009", "intended_use": "Treatment of 5-fluorouracil overdose", "eu_designation_number": "EU/3/09/637", "status": "Positive", "first_published_date": "09/06/2009", "last_updated_date": "11/09/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-637" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "4-Amino-(6R,S)-5,6,7,8-tetrahydro-L-biopterin dihydrochloride (ronopterin)", "date_of_designation_or_refusal": "28/08/2006", "intended_use": "Treatment of moderate and severe traumatic brain injury", "eu_designation_number": "EU/3/06/390", "status": "Positive", "first_published_date": "24/02/2009", "last_updated_date": "11/08/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-06-390" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "adeno-associated virus serotype HSC 15 expressing human iduronate 2-sulfatase (Bevufenogene nofeparvovec)", "date_of_designation_or_refusal": "16/03/2022", "intended_use": "Treatment of mucopolysaccharidosis type II (Hunter Syndrome)", "eu_designation_number": "EU/3/22/2592", "status": "Withdrawn", "first_published_date": "20/06/2022", "last_updated_date": "08/08/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2592" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "adeno-associated virus serotype HSC15 expressing human phenylalanine hydroxylase", "date_of_designation_or_refusal": "14/12/2018", "intended_use": "Treatment of phenylalanine hydroxylase deficiency", "eu_designation_number": "EU/3/18/2109", "status": "Withdrawn", "first_published_date": "08/03/2019", "last_updated_date": "08/08/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2109" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Human telomerase reverse transcriptase peptide (611-626) (tertomotide)", "date_of_designation_or_refusal": "25/07/2006", "intended_use": "Treatment of pancreatic cancer", "eu_designation_number": "EU/3/06/384", "status": "Withdrawn", "first_published_date": "21/09/2009", "last_updated_date": "08/08/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-06-384" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Humanised IgG1 monoclonal antibody against SEZ6 linked to N-acetyl-calicheamicin", "date_of_designation_or_refusal": "15/10/2021", "intended_use": "Treatment of small cell lung cancer", "eu_designation_number": "EU/3/21/2502", "status": "Withdrawn", "first_published_date": "20/01/2022", "last_updated_date": "31/07/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2502" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "tobramycin", "date_of_designation_or_refusal": "17/03/2003", "intended_use": "Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis", "eu_designation_number": "EU/3/03/140", "status": "Expired", "first_published_date": "07/03/2007", "last_updated_date": "31/07/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-03-140" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Itraconazole", "date_of_designation_or_refusal": "23/08/2017", "intended_use": "Treatment of naevoid basal-cell carcinoma syndrome (Gorlin syndrome)", "eu_designation_number": "EU/3/17/1901", "status": "Withdrawn", "first_published_date": "17/10/2017", "last_updated_date": "27/07/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1901" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Fusion protein composed of the first 2 immunoglobulin-like domains of the human Roundabout guidance receptor 2 fused to a human IgG1 crystallised fragment", "date_of_designation_or_refusal": "16/05/2022", "intended_use": "Treatment of focal segmental glomerulosclerosis", "eu_designation_number": "EU/3/22/2611", "status": "Withdrawn", "first_published_date": "14/12/2022", "last_updated_date": "27/07/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2611" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Iodine (124I) evuzamitide", "date_of_designation_or_refusal": "09/12/2022", "intended_use": "Diagnosis of ATTR amyloidosis", "eu_designation_number": "EU/3/22/2725", "status": "Positive", "first_published_date": "13/03/2023", "last_updated_date": "25/07/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2725" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Iodine (124I) evuzamitide", "date_of_designation_or_refusal": "11/11/2022", "intended_use": "Diagnosis of AL amyloidosis", "eu_designation_number": "EU/3/22/2712", "status": "Positive", "first_published_date": "23/01/2023", "last_updated_date": "25/07/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2712" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "codon-optimised human cystic fibrosis transmembrane conductance regulator messenger ribonucleic acid complexed with lipid-based nanoparticles;pomulmeran", "date_of_designation_or_refusal": "01/04/2019", "intended_use": "Treatment of cystic fibrosis", "eu_designation_number": "EU/3/19/2150", "status": "Withdrawn", "first_published_date": "07/05/2019", "last_updated_date": "21/07/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2150" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "adeno-associated virus serotype HSC15 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3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib)", "date_of_designation_or_refusal": "02/02/2010", "intended_use": "Treatment of acute lymphoblastic leukaemia", "eu_designation_number": "EU/3/09/715", "status": "Expired", "first_published_date": "04/03/2010", "last_updated_date": "20/07/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-715" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib)", "date_of_designation_or_refusal": "02/02/2010", "intended_use": "Treatment of chronic myeloid leukaemia", "eu_designation_number": "EU/3/09/716", "status": "Expired", "first_published_date": "04/03/2010", "last_updated_date": "20/07/2023", "orphan_designation_url": 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"orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-06-412" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "5'-ASCSASTSCSASGSTSCSTSGSASUSASASGSCSTSA-3' (lademirsen)", "date_of_designation_or_refusal": "19/03/2015", "intended_use": "Treatment of Alport syndrome", "eu_designation_number": "EU/3/15/1451", "status": "Withdrawn", "first_published_date": "11/05/2015", "last_updated_date": "05/06/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1451" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "viltolarsen", "date_of_designation_or_refusal": "04/06/2020", "intended_use": "Treatment of Duchenne muscular dystrophy", "eu_designation_number": "EU/3/20/2282", "status": "Positive", "first_published_date": "19/08/2020", "last_updated_date": "01/06/2023", "orphan_designation_url": 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-1994" }, { "medicine_name": "Tibsovo; Tidhesco", "related_ema_product_number": "EMEA/H/C/005936; EMEA/H/C/006174", "active_substance": "Ivosidenib", "date_of_designation_or_refusal": "12/12/2016", "intended_use": "Treatment of acute myeloid leukaemia", "eu_designation_number": "EU/3/16/1802", "status": "Positive", "first_published_date": "09/01/2017", "last_updated_date": "12/05/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1802" }, { "medicine_name": "Nugalviq", "related_ema_product_number": "EMEA/H/C/006270", "active_substance": "govorestat", "date_of_designation_or_refusal": "21/06/2022", "intended_use": "Treatment of galactosaemia", "eu_designation_number": "EU/3/22/2642", "status": "Positive", "first_published_date": "22/09/2022", "last_updated_date": "10/05/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2642" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "pegunigalsidase alfa", "date_of_designation_or_refusal": "12/12/2017", "intended_use": "Treatment of Fabry disease", "eu_designation_number": "EU/3/17/1953", "status": "Withdrawn", "first_published_date": "23/01/2018", "last_updated_date": "08/05/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1953" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "N-[(2S)-5-{[(1R, 2S)-2-(4-fluorophenyl)cyclopropyl]amino}-1-(4-methylpiperazin-1-yl)-1-oxopentan-2-yl]-4-(1H-1,2,3-triazol-1-yl)benzamide, bis-tosilate salt (Bomedemstat ditosilate)", "date_of_designation_or_refusal": "14/10/2016", "intended_use": "Treatment of myelofibrosis", "eu_designation_number": "EU/3/16/1757", "status": "Positive", "first_published_date": "15/11/2016", "last_updated_date": "08/05/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1757" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "troriluzole hydrochloride", "date_of_designation_or_refusal": "10/12/2021", "intended_use": "Treatment of spinocerebellar ataxia", "eu_designation_number": "EU/3/21/2553", "status": "Positive", "first_published_date": "05/05/2022", "last_updated_date": "08/05/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2553" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "rozanolixizumab", "date_of_designation_or_refusal": "11/01/2019", "intended_use": "Treatment of immune thrombocytopenia", "eu_designation_number": "EU/3/18/2131", "status": "Withdrawn", "first_published_date": "27/03/2019", "last_updated_date": "08/05/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2131" }, { "medicine_name": "Oxbryta", "related_ema_product_number": "EMEA/H/C/004869", "active_substance": "2-hydroxy-6-((2-(1-isopropyl-1H-pyrazol-5-yl)pyridin-3-yl)methoxy)benzaldehyde (voxelotor)", "date_of_designation_or_refusal": "18/11/2016", "intended_use": "Treatment of sickle cell disease", "eu_designation_number": "EU/3/16/1769", "status": "Positive", "first_published_date": "14/12/2016", "last_updated_date": "04/05/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1769" }, { "medicine_name": "SomaKit TOC", "related_ema_product_number": "EMEA/H/C/004140", "active_substance": "Gallium (68Ga)-edotreotide", "date_of_designation_or_refusal": "19/03/2015", "intended_use": "Diagnosis of gastro-entero-pancreatic neuroendocrine tumours", "eu_designation_number": "EU/3/15/1450", "status": "Positive", "first_published_date": "11/05/2015", "last_updated_date": "03/05/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1450" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "68Ga-DOTA-pABzA-DIG-dPhe-Gln-Trp-Ala-Val-Gly-His-NHCH[(CH2-CH(CH3)2]2", "date_of_designation_or_refusal": "12/12/2016", "intended_use": "Diagnosis of gastrointestinal stromal tumours", "eu_designation_number": "EU/3/16/1794", "status": "Positive", "first_published_date": "09/01/2017", "last_updated_date": "03/05/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1794" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Gallium [Ga-68]-N-[(4,7,10-Tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl-Lcysteinyl-L-threonine-cyclic(2-7)disulfide", "date_of_designation_or_refusal": "19/02/2014", "intended_use": "Diagnosis of gastro-entero-pancreatic neuroendocrine tumours", "eu_designation_number": "EU/3/14/1237", "status": "Positive", "first_published_date": "01/04/2014", "last_updated_date": "03/05/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1237" }, { "medicine_name": "Lutathera", "related_ema_product_number": "EMEA/H/C/004123", "active_substance": "Lutetium (177Lu)-N-[(4,7,10-Tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl-L-cysteinyl-L-threonine-cyclic(2-7)disulfide (lutetium (177Lu) oxodotreotide)", "date_of_designation_or_refusal": "31/01/2008", "intended_use": "Treatment of gastro-entero-pancreatic neuroendocrine tumours", "eu_designation_number": "EU/3/07/523", "status": "Positive", "first_published_date": "10/07/2008", "last_updated_date": "03/05/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-523" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Bomedemstat ditosilate", "date_of_designation_or_refusal": "21/06/2021", "intended_use": "Treatment of essential thrombocythaemia", "eu_designation_number": "EU/3/21/2451", "status": "Positive", "first_published_date": "11/05/2022", "last_updated_date": "02/05/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2451" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "ex-vivo fused autologous human bone marrow-derived mesenchymal stem cell with allogenic human myoblast", "date_of_designation_or_refusal": "31/07/2018", "intended_use": "Treatment of Duchenne muscular dystrophy", "eu_designation_number": "EU/3/18/2045", "status": "Positive", "first_published_date": "10/10/2018", "last_updated_date": "28/04/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2045" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Ex vivo fused normal allogeneic human myoblast with another normal allogeneic human myoblast", "date_of_designation_or_refusal": "19/11/2018", "intended_use": "Treatment of Duchenne muscular dystrophy", "eu_designation_number": "EU/3/18/2088", "status": "Positive", "first_published_date": "25/02/2019", "last_updated_date": "28/04/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2088" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Ile-Ser-Ile-Thr-Glu-Ile-Lys-Gly-Val-Ile-Val-His-Arg-Ile-Glu-Thr-Ile-Leu-Phe-Lys-Lys-Lys-Lys-Glu-Met-Pro-Ser-Glu-Glu-Gly-Tyr-Gln-Asp", "date_of_designation_or_refusal": "19/11/2018", "intended_use": "Treatment of multiple system atrophy", "eu_designation_number": "EU/3/18/2095", "status": "Positive", "first_published_date": "25/02/2019", "last_updated_date": "28/04/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2095" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Ex vivo fused normal allogeneic human myoblast with autologous human myoblast derived from Duchenne muscular dystrophy affected donor", "date_of_designation_or_refusal": "19/11/2019", "intended_use": "Treatment of Duchenne muscular dystrophy", "eu_designation_number": "EU/3/18/2089", "status": "Positive", "first_published_date": "25/02/2019", "last_updated_date": "28/04/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2089" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "tazemetostat", "date_of_designation_or_refusal": "21/03/2018", "intended_use": "Treatment of follicular lymphoma", "eu_designation_number": "EU/3/18/2005", "status": "Positive", "first_published_date": "15/05/2018", "last_updated_date": "28/04/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2005" }, { "medicine_name": "Kygevvi", "related_ema_product_number": "EMEA/H/C/005119", "active_substance": "thymidine;deoxycytidine", "date_of_designation_or_refusal": "20/04/2017", "intended_use": "Treatment of mitochondrial DNA depletion syndrome, myopathic form", "eu_designation_number": "EU/3/17/1870", "status": "Positive", "first_published_date": "15/05/2017", "last_updated_date": "25/04/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1870" }, { "medicine_name": "Fintepla", "related_ema_product_number": "EMEA/H/C/003933", "active_substance": "fenfluramine hydrochloride", "date_of_designation_or_refusal": "27/02/2017", "intended_use": "Treatment of Lennox-Gastaut syndrome", "eu_designation_number": "EU/3/17/1836", "status": "Positive", "first_published_date": "31/03/2017", "last_updated_date": "25/04/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1836" }, { "medicine_name": "Fintepla", "related_ema_product_number": "EMEA/H/C/003933", "active_substance": "fenfluramine hydrochloride", "date_of_designation_or_refusal": "18/12/2013", "intended_use": "Treatment of Dravet syndrome", "eu_designation_number": "EU/3/13/1219", "status": "Positive", "first_published_date": "23/01/2014", "last_updated_date": "25/04/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1219" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "3-(3-(3,5-bis(trifluoromethyl)phenyl)-1h-pyrazol-1-yl)propanoic acid", "date_of_designation_or_refusal": "24/04/2019", "intended_use": "Treatment of Stargardt's disease", "eu_designation_number": "EU/3/19/2152", "status": "Withdrawn", "first_published_date": "14/06/2019", "last_updated_date": "13/04/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2152" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "tripotassium citrate monohydrate;potassium hydrogen carbonate", "date_of_designation_or_refusal": "09/01/2020", "intended_use": "Treatment of cystinuria", "eu_designation_number": "EU/3/19/2239", "status": "Positive", "first_published_date": "19/05/2020", "last_updated_date": "13/04/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2239" }, { "medicine_name": "Miplyffa", "related_ema_product_number": "EMEA/H/C/005203", "active_substance": "arimoclomol citrate", "date_of_designation_or_refusal": "19/11/2014", "intended_use": "Treatment of Niemann-Pick's disease, type C", "eu_designation_number": "EU/3/14/1376", "status": "Positive", "first_published_date": "16/01/2015", "last_updated_date": "11/04/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1376" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "(R)-1-[1-(4-acetoxy-3,3-dimethyl-2-oxo-butyl)-2-oxo-5-(pyridin-2-yl)-2,3-dihydro-1H-benzo[e][1,4]diazepin-3-yl]-3-(3-methylamino-phenyl)-urea (ceclazepide)", "date_of_designation_or_refusal": "14/12/2015", "intended_use": "Treatment of gastro-entero-pancreatic neuroendocrine tumours", "eu_designation_number": "EU/3/15/1588", "status": "Withdrawn", "first_published_date": "02/02/2016", "last_updated_date": "04/04/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1588" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "streptococcus pyogenes, group A, type 3, strain Su, inactivated", "date_of_designation_or_refusal": "16/05/2022", "intended_use": "Treatment of lymphatic malformations", "eu_designation_number": "EU/3/22/2613", "status": "Positive", "first_published_date": "14/12/2022", "last_updated_date": "04/04/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2613" }, { "medicine_name": "Qalsody", "related_ema_product_number": "EMEA/H/C/005493", "active_substance": "synthetic ribonucleic acid oligonucleotide directed against superoxide dismutase 1 messenger ribonucleic acid (tofersen)", "date_of_designation_or_refusal": "29/08/2016", "intended_use": "Treatment of amyotrophic lateral sclerosis", "eu_designation_number": "EU/3/16/1732", "status": "Positive", "first_published_date": "03/10/2016", "last_updated_date": "03/04/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1732" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Humanised anti-alpha ν beta 6 monoclonal antibody", "date_of_designation_or_refusal": "29/07/2014", "intended_use": "Treatment of idiopathic pulmonary fibrosis", "eu_designation_number": "EU/3/14/1301", "status": "Positive", "first_published_date": "01/09/2014", "last_updated_date": "03/04/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1301" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "autologous CD34+ cells transduced with a lentiviral vector containing the human ADA gene", "date_of_designation_or_refusal": "07/06/2013", "intended_use": "Treatment of adenosine-deaminase-deficient severe combined immunodeficiency", "eu_designation_number": "EU/3/13/1134", "status": "Positive", "first_published_date": "25/06/2013", "last_updated_date": "03/04/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1134" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Iodine (131I) iobenguane", "date_of_designation_or_refusal": "31/01/2008", "intended_use": "Treatment of neuroblastoma", "eu_designation_number": "EU/3/07/525", "status": "Positive", "first_published_date": "10/07/2008", "last_updated_date": "29/03/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-525" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "recombinant adeno-associated viral vector containing the human RPGR gene", "date_of_designation_or_refusal": "30/05/2016", "intended_use": "Treatment of retinitis pigmentosa caused by mutations in the RPGR gene", "eu_designation_number": "EU/3/16/1665", "status": "Positive", "first_published_date": "22/06/2016", "last_updated_date": "27/03/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1665" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "(S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidine-3-yl)chroman-2-carboxamide hydrochloride", "date_of_designation_or_refusal": "28/06/2019", "intended_use": "Treatment of maternally-inherited diabetes and deafness", "eu_designation_number": "EU/3/19/2168", "status": "Withdrawn", "first_published_date": "02/10/2019", "last_updated_date": "27/03/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2168" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "(S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride", "date_of_designation_or_refusal": "10/08/2015", "intended_use": "Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes", "eu_designation_number": "EU/3/15/1543", "status": "Withdrawn", "first_published_date": "02/10/2015", "last_updated_date": "27/03/2023", "orphan_designation_url": 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"status": "Positive", "first_published_date": "13/03/2023", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2726" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "16-base single stranded RNA targeting miR-23b linked to oleic acid", "date_of_designation_or_refusal": "09/12/2022", "intended_use": "Treatment of myotonic disorders", "eu_designation_number": "EU/3/22/2724", "status": "Positive", "first_published_date": "13/03/2023", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2724" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Lentiviral vector containing the human MYO7A gene", "date_of_designation_or_refusal": "23/03/2010", "intended_use": "Treatment of retinitis pigmentosa in Usher syndrome 1B", "eu_designation_number": "EU/3/10/727", "status": "Positive", "first_published_date": "26/03/2010", "last_updated_date": "13/03/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-727" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Lentiviral vector containing the human ABCA4 gene", "date_of_designation_or_refusal": "02/02/2010", "intended_use": "Treatment of Stargardt's disease", "eu_designation_number": "EU/3/09/720", "status": "Positive", "first_published_date": "04/03/2010", "last_updated_date": "13/03/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-720" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "clofazimine", "date_of_designation_or_refusal": "21/08/2019", "intended_use": "Treatment of nontuberculous mycobacterial lung disease", "eu_designation_number": "EU/3/19/2200", "status": "Positive", "first_published_date": "16/01/2020", "last_updated_date": 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{ "medicine_name": "", "related_ema_product_number": "", "active_substance": "cannabidiol", "date_of_designation_or_refusal": "25/02/2022", "intended_use": "Treatment of fragile X syndrome (FXS)", "eu_designation_number": "EU/3/22/2583", "status": "Positive", "first_published_date": "13/05/2022", "last_updated_date": "07/03/2023", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2583" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "3,5-diamino-6-chloro-N-(N-(4-(4-(2-(hexyl((2S,3R,4R,5R)-2,3,4,5,6-pentahydroxyhexyl)amino)ethoxy)phenyl)butyl)-carbamimidoyl)pyrazine-2-carboxamide, Sodium chloride solution 4.2% (w/v)", "date_of_designation_or_refusal": "16/11/2020", "intended_use": "Treatment of primary ciliary dyskinesia", "eu_designation_number": "EU/3/20/2363", "status": "Positive", "first_published_date": "15/02/2021", "last_updated_date": "07/03/2023", "orphan_designation_url": 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"related_ema_product_number": "", "active_substance": "3-[4-(1H-imidazol-1-ylmethyl)phenyl]-5-(2-methylpropyl)thiophene-2-[(N-butyloxylcarbamate)-sulphonamide] sodium salt", "date_of_designation_or_refusal": "14/07/2016", "intended_use": "Treatment of idiopathic pulmonary fibrosis", "eu_designation_number": "EU/3/16/1692", "status": "Positive", "first_published_date": "06/09/2016", "last_updated_date": "08/12/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1692" }, { "medicine_name": "Pyrukynd", "related_ema_product_number": "EMEA/H/C/005540", "active_substance": "mitapivat sulfate", "date_of_designation_or_refusal": "22/04/2020", "intended_use": "Treatment of pyruvate kinase deficiency", "eu_designation_number": "EU/3/20/2270", "status": "Positive", "first_published_date": "12/08/2020", "last_updated_date": "07/12/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2270" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "allogeneic retinal pigment epithelial cells genetically modified with a non-viral vector to express beta-domain deleted human factor VIII", "date_of_designation_or_refusal": "09/12/2020", "intended_use": "Treatment of haemophilia A", "eu_designation_number": "EU/3/20/2380", "status": "Withdrawn", "first_published_date": "27/05/2021", "last_updated_date": "05/12/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2380" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Lentiviral vector encoding human coagulation factor IX", "date_of_designation_or_refusal": "26/02/2019", "intended_use": "Treatment of haemophilia B", "eu_designation_number": "EU/3/19/2141", "status": "Positive", "first_published_date": "09/04/2019", "last_updated_date": "02/12/2022", "orphan_designation_url": 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"active_substance": "Linerixibat", "date_of_designation_or_refusal": "12/11/2021", "intended_use": "Treatment of primary biliary cholangitis", "eu_designation_number": "EU/3/21/2515", "status": "Positive", "first_published_date": "07/06/2022", "last_updated_date": "29/11/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2515" }, { "medicine_name": "Livtencity", "related_ema_product_number": "EMEA/H/C/005787", "active_substance": "Maribavir", "date_of_designation_or_refusal": "07/06/2013", "intended_use": "Treatment of cytomegalovirus disease in patients with impaired cell-mediated immunity", "eu_designation_number": "EU/3/13/1133", "status": "Positive", "first_published_date": "25/06/2013", "last_updated_date": "24/11/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1133" }, { "medicine_name": "Livtencity", "related_ema_product_number": "EMEA/H/C/005787", "active_substance": "Maribavir", "date_of_designation_or_refusal": "18/12/2007", "intended_use": "Prevention of cytomegalovirus disease in patients with impaired cell-mediated immunity", "eu_designation_number": "EU/3/07/519", "status": "Positive", "first_published_date": "23/09/2009", "last_updated_date": "24/11/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-519" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "1-(2-hydroxyethyl)-8-{[5-(4-methylpiperazin-1-yl)-2-(trifluoromethoxy) phenyl]amino}-4,5-dihydro-1H-pyrazolo[4,3-h]quinazoline-3-carboxamide fumarate salt (onvansertib)", "date_of_designation_or_refusal": "24/08/2018", "intended_use": "Treatment of acute myeloid leukaemia", "eu_designation_number": "EU/3/18/2057", "status": "Withdrawn", "first_published_date": "19/12/2018", "last_updated_date": "22/11/2022", "orphan_designation_url": 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1026" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "recombinant chimeric monoclonal antibody against CD20 (ublituximab)", "date_of_designation_or_refusal": "26/11/2009", "intended_use": "Treatment of chronic lymphocytic leukaemia", "eu_designation_number": "EU/3/09/699", "status": "Positive", "first_published_date": "09/12/2009", "last_updated_date": "22/11/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-699" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "6alpha-ethyl-chenodeoxycholic acid", "date_of_designation_or_refusal": "27/07/2010", "intended_use": "Treatment of primary biliary cirrhosis", "eu_designation_number": "EU/3/10/753", "status": "Withdrawn", "first_published_date": "05/08/2010", "last_updated_date": "21/11/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-753" }, { "medicine_name": "Loargys", "related_ema_product_number": "EMEA/H/C/005484", "active_substance": "poly(oxy-1,2-ethanediyl), alpha-(carboxymethyl)-omega-methoxy-, amide with arginase 1 [cobalt cofactor] (synthetic human) (1:10), trimer (pegzilarginase)", "date_of_designation_or_refusal": "14/07/2016", "intended_use": "Treatment of hyperargininaemia", "eu_designation_number": "EU/3/16/1701", "status": "Positive", "first_published_date": "06/09/2016", "last_updated_date": "18/11/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1701" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Glyceryl tri-(4-phenylbutyrate)", "date_of_designation_or_refusal": "10/06/2010", "intended_use": "Treatment of citrullinaemia type 2", "eu_designation_number": "EU/3/10/739", "status": "Positive", "first_published_date": "24/06/2010", 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-997" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "[Gly2]-recombinant human glucagon-like peptide", "date_of_designation_or_refusal": "12/12/2001", "intended_use": "Treatment of short bowel syndrome", "eu_designation_number": "EU/3/01/077", "status": "Positive", "first_published_date": "15/05/2009", "last_updated_date": "12/10/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-01-077" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "4-hydroxy-2,2,6,6-tetramethylpiperidine-N-oxyl", "date_of_designation_or_refusal": "12/12/2017", "intended_use": "Treatment of familial cerebral cavernous malformations", "eu_designation_number": "EU/3/17/1948", "status": "Positive", "first_published_date": "23/01/2018", "last_updated_date": "11/10/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1948" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Adeno-associated viral vector serotype rh10 containing the human factor IX gene for the treatment of haemophilia B-", "date_of_designation_or_refusal": "14/12/2015", "intended_use": "Treatment of haemophilia B", "eu_designation_number": "EU/3/15/1599", "status": "Withdrawn", "first_published_date": "03/02/2016", "last_updated_date": "11/10/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1599" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "recombinant adeno-associated viral vector containing the human retinoschisin gene", "date_of_designation_or_refusal": "12/03/2013", "intended_use": "Treatment of X-linked juvenile retinoschisis", "eu_designation_number": "EU/3/13/1107", "status": "Positive", "first_published_date": "05/04/2013", "last_updated_date": "06/10/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1107" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "recombinant adeno-associated viral vector containing human alpha-1 antitrypsin gene", "date_of_designation_or_refusal": "20/03/2007", "intended_use": "Treatment of congenital alpha-1 antitrypsin deficiency", "eu_designation_number": "EU/3/07/440", "status": "Positive", "first_published_date": "19/07/2007", "last_updated_date": "06/10/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-440" }, { "medicine_name": "Omjjara", "related_ema_product_number": "EMEA/H/C/005768", "active_substance": "N-(cyanomethyl)-4-(2-{[4-(morpholin-4-yl)phenyl]amino}pyrimidin-4-yl)benzamide, dihydrochloride salt (momelotinib)", "date_of_designation_or_refusal": "05/08/2011", "intended_use": "Treatment of primary myelofibrosis", "eu_designation_number": "EU/3/11/888", "status": "Positive", "first_published_date": "16/08/2011", "last_updated_date": "04/10/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-888" }, { "medicine_name": "Omjjara", "related_ema_product_number": "EMEA/H/C/005768", "active_substance": "N-(cyanomethyl)-4-(2-{[4-(morpholin-4-yl)phenyl]amino}pyrimidin-4-yl)benzamide, dihydrochloride salt (momelotinib)", "date_of_designation_or_refusal": "05/08/2011", "intended_use": "Treatment of post-polycythaemia vera myelofibrosis", "eu_designation_number": "EU/3/11/886", "status": "Positive", "first_published_date": "16/08/2011", "last_updated_date": "04/10/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-886" }, { "medicine_name": "Omjjara", "related_ema_product_number": "EMEA/H/C/005768", "active_substance": "N-(cyanomethyl)-4-(2-{[4-(morpholin-4-yl)phenyl]amino}pyrimidin-4-yl)benzamide, dihydrochloride salt (momelotinib)", "date_of_designation_or_refusal": "05/08/2011", "intended_use": "Treatment of post-essential thrombocythaemia myelofibrosis", "eu_designation_number": "EU/3/11/887", "status": "Positive", "first_published_date": "16/08/2011", "last_updated_date": "04/10/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-887" }, { "medicine_name": "Takhzyro", "related_ema_product_number": "EMEA/H/C/004806", "active_substance": "recombinant human IgG1 kappa light chain monoclonal antibody targeting plasma kallikrein (lanadelumab)", "date_of_designation_or_refusal": "09/10/2015", "intended_use": "Treatment of hereditary angioedema", "eu_designation_number": "EU/3/15/1551", "status": "Positive", "first_published_date": "16/11/2015", "last_updated_date": "04/10/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1551" }, { "medicine_name": "Natpar", "related_ema_product_number": "EMEA/H/C/003861", "active_substance": "recombinant human parathyroid hormone", "date_of_designation_or_refusal": "16/01/2014", "intended_use": "Treatment of hypoparathyroidism", "eu_designation_number": "EU/3/13/1210", "status": "Positive", "first_published_date": "23/01/2014", "last_updated_date": "04/10/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1210" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "recombinant human arylsulfatase A", "date_of_designation_or_refusal": "26/11/2010", "intended_use": "treatment of metachromatic leukodystrophy", "eu_designation_number": "EU/3/10/813", "status": "Positive", "first_published_date": "17/12/2010", "last_updated_date": "04/10/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-813" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "(S)-3-((3-(1-((6-(3,4-dimethoxyphenyl)pryazin-2-yl)amino)ethyl)phenyl)carbamoyl)-5-methylpridin-1-ium (seralutinib)", "date_of_designation_or_refusal": "29/05/2019", "intended_use": "Treatment of pulmonary arterial hypertension", "eu_designation_number": "EU/3/19/2158", "status": "Positive", "first_published_date": "09/08/2019", "last_updated_date": "30/09/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2158" }, { "medicine_name": "Imcivree", "related_ema_product_number": "EMEA/H/C/005089", "active_substance": "setmelanotide", "date_of_designation_or_refusal": "21/08/2019", "intended_use": "Treatment of Bardet-Biedl syndrome", "eu_designation_number": "EU/3/19/2192", "status": "Positive", "first_published_date": "14/01/2020", "last_updated_date": "30/09/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2192" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "variant of recombinant human fibroblast growth factor 19", "date_of_designation_or_refusal": "14/12/2015", "intended_use": "Treatment of primary sclerosing cholangitis", "eu_designation_number": "EU/3/15/1584", "status": "Withdrawn", "first_published_date": "02/02/2016", "last_updated_date": "28/09/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1584" }, { "medicine_name": "Roctavian; Roctavian", "related_ema_product_number": "EMEA/H/C/004749; EMEA/H/C/005830", "active_substance": "adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene", "date_of_designation_or_refusal": "21/03/2016", "intended_use": "Treatment of haemophilia A", "eu_designation_number": "EU/3/16/1622", "status": "Positive", "first_published_date": "04/05/2016", "last_updated_date": "28/09/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1622" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Combretastatin A1 diphosphate", "date_of_designation_or_refusal": "14/12/2015", "intended_use": "Treatment of acute myeloid leukaemia", "eu_designation_number": "EU/3/15/1587", "status": "Positive", "first_published_date": "02/02/2016", "last_updated_date": "28/09/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1587" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Ilixadencel", "date_of_designation_or_refusal": "26/03/2021", "intended_use": "Treatment of gastrointestinal stromal tumours", "eu_designation_number": "EU/3/21/2418", "status": "Positive", "first_published_date": "19/01/2022", "last_updated_date": "28/09/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2418" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "autologous CD34+ cells transduced with lentiviral vector encoding the human beta globin gene", "date_of_designation_or_refusal": "30/05/2016", "intended_use": "Treatment of beta thalassaemia intermedia and major", "eu_designation_number": "EU/3/16/1660", "status": "Positive", "first_published_date": "23/06/2016", "last_updated_date": "26/09/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1660" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "ofranergene obadenovec", "date_of_designation_or_refusal": "16/10/2017", "intended_use": "Treatment of ovarian cancer", "eu_designation_number": "EU/3/17/1926", "status": "Withdrawn", "first_published_date": "08/01/2018", "last_updated_date": "26/09/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1926" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "adenovirus-associated vector containing human Fas-c gene (ofranergene obadenovec)", "date_of_designation_or_refusal": "06/06/2012", "intended_use": "Treatment of glioma", "eu_designation_number": "EU/3/12/1002", "status": "Withdrawn", "first_published_date": "04/07/2012", "last_updated_date": "26/09/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1002" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "modified mRNA encoding human methylmalonyl-coenzyme A mutase containing a polymorphism at position 671", "date_of_designation_or_refusal": "21/06/2022", "intended_use": "Treatment of methylmalonic acidaemia", "eu_designation_number": "EU/3/22/2638", "status": "Positive", "first_published_date": "22/09/2022", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2638" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl -(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-P-thioadenylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-P-thioguanylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl", "date_of_designation_or_refusal": "21/06/2022", "intended_use": "Treatment of cystic fibrosis", "eu_designation_number": "EU/3/22/2639", "status": "Positive", "first_published_date": "22/09/2022", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2639" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "human allogeneic keratinocytes", "date_of_designation_or_refusal": "21/06/2022", "intended_use": "Treatment of partial deep dermal and full thickness burns", "eu_designation_number": "EU/3/22/2637", "status": "Positive", "first_published_date": "22/09/2022", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2637" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "2'-O-(2-methoxyethyl) modified antisense oligonucleotide targeting UBE3A antisense transcript RNA", "date_of_designation_or_refusal": "21/06/2022", "intended_use": "Treatment of Angelman syndrome", "eu_designation_number": "EU/3/22/2636", "status": "Positive", "first_published_date": "22/09/2022", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2636" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "epcoritamab", "date_of_designation_or_refusal": "21/06/2022", "intended_use": "Treatment of follicular lymphoma", "eu_designation_number": "EU/3/22/2634", "status": "Withdrawn", "first_published_date": "22/09/2022", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2634" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "sirolimus", "date_of_designation_or_refusal": "21/06/2022", "intended_use": "Treatment of lymphatic malformations", "eu_designation_number": "EU/3/22/2633", "status": "Positive", "first_published_date": "22/09/2022", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2633" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Lithium carbonate", "date_of_designation_or_refusal": "21/06/2022", "intended_use": "treatment of TBR1-related disorder", "eu_designation_number": "EU/3/22/2632", "status": "Positive", "first_published_date": "22/09/2022", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2632" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "allogenic cytomegalovirus-specific cytotoxic T lymphocytes (adimlecleucel)", "date_of_designation_or_refusal": "18/11/2016", "intended_use": "Treatment of cytomegalovirus infection in patients with impaired cell-mediated immunity", "eu_designation_number": "EU/3/16/1773", "status": "Withdrawn", "first_published_date": "13/12/2016", "last_updated_date": "16/09/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1773" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "allogeneic CD34+ cells expanded ex vivo with an aryl hydrocarbon receptor antagonist (spanlecortemlocel)", "date_of_designation_or_refusal": "16/12/2014", "intended_use": "Treatment of acute lymphoblastic leukaemia", "eu_designation_number": "EU/3/14/1382", "status": "Withdrawn", "first_published_date": "26/02/2015", "last_updated_date": "16/09/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1382" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "autologous CD34+ cells transduced ex vivo with a lentiviral vector containing a modified gamma-globin gene", "date_of_designation_or_refusal": "13/11/2020", "intended_use": "Treatment of sickle cell disease", "eu_designation_number": "EU/3/20/2356", "status": "Withdrawn", "first_published_date": "15/02/2021", "last_updated_date": "16/09/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2356" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "self-complementary adeno-associated viral vector serotype 9 containing the SGSH gene", "date_of_designation_or_refusal": "20/09/2016", "intended_use": "Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome)", "eu_designation_number": "EU/3/16/1761", "status": "Positive", "first_published_date": "15/11/2016", "last_updated_date": "16/09/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1761" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "sodium (4Z,7Z,10R,11E,13E,15Z,17S,19Z)10,17-dihydroxy-docosa-4,7,11,13,15,19-hexaenoate", "date_of_designation_or_refusal": "21/06/2022", "intended_use": "Prevention of retinopathy of prematurity", "eu_designation_number": "EU/3/22/2631", "status": "Positive", "first_published_date": "15/09/2022", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2631" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Pirfenidone", "date_of_designation_or_refusal": "24/06/2022", "intended_use": "Treatment of idiopathic pulmonary fibrosis", "eu_designation_number": "EU/3/22/2630", "status": "Positive", "first_published_date": "15/09/2022", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2630" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "4-[[(3S)-1-benzylpyrrolidin-3-yl]-methylamino]-2-fluoro-5-methyl-N-(1,3-thiazol-4-yl)benzenesulfonamide", "date_of_designation_or_refusal": "21/06/2022", "intended_use": "Treatment of SCN8A developmental and epileptic encephalopathy", 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"eu_designation_number": "EU/3/22/2627", "status": "Positive", "first_published_date": "15/09/2022", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2627" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "allogeneic human dendritic cells derived from a CD34+ progenitor cell line", "date_of_designation_or_refusal": "22/05/2012", "intended_use": "Treatment of acute myeloid leukaemia", "eu_designation_number": "EU/3/12/969", "status": "Positive", "first_published_date": "06/07/2012", "last_updated_date": "15/09/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-969" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "allogeneic faecal microbiota, pooled", "date_of_designation_or_refusal": "19/11/2018", "intended_use": "Treatment of graft-versus-host disease", "eu_designation_number": "EU/3/18/2083", 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"Xegafri", "related_ema_product_number": "EMEA/H/C/004053", "active_substance": "sirolimus", "date_of_designation_or_refusal": "30/08/2011", "intended_use": "Treatment of chronic non-infectious uveitis", "eu_designation_number": "EU/3/11/898", "status": "Withdrawn", "first_published_date": "21/09/2011", "last_updated_date": "30/08/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-898" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "human monoclonal antibody against human interleukin 13", "date_of_designation_or_refusal": "13/11/2013", "intended_use": "Treatment of eosinophilic oesophagitis", "eu_designation_number": "EU/3/13/1205", "status": "Withdrawn", "first_published_date": "02/12/2013", "last_updated_date": "30/08/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1205" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Melphalan flufenamide", "date_of_designation_or_refusal": "19/03/2015", "intended_use": "Treatment of plasma cell myeloma", "eu_designation_number": "EU/3/15/1463", "status": "Withdrawn", "first_published_date": "11/05/2015", "last_updated_date": "22/08/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1463" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "autologous dendritic cells pulsed with killed ovarian cancer cells and matured by TLR3 ligand ex vivo", "date_of_designation_or_refusal": "16/04/2018", "intended_use": "Treatment of ovarian cancer", "eu_designation_number": "EU/3/18/2009", "status": "Withdrawn", "first_published_date": "17/05/2018", "last_updated_date": "17/08/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2009" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "recombinant truncated N-terminal fragment of human lens epithelium-derived growth factor", "date_of_designation_or_refusal": "23/08/2017", "intended_use": "Treatment of retinitis pigmentosa", "eu_designation_number": "EU/3/17/1908", "status": "Withdrawn", "first_published_date": "17/10/2017", "last_updated_date": "12/08/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1908" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "allogeneic peripheral blood mononuclear cells incubated ex-vivo with 16, 16-dimethyl prostaglandin E2;dexamethasone", "date_of_designation_or_refusal": "18/11/2016", "intended_use": "Treatment in haematopoietic stem cell transplantation", "eu_designation_number": "EU/3/16/1774", "status": "Withdrawn", "first_published_date": "13/12/2016", "last_updated_date": "10/08/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1774" 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"02/07/2008", "last_updated_date": "09/08/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-486" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "adenovirus associated viral vector serotype 4 containing the human RPE65 gene", "date_of_designation_or_refusal": "22/10/2007", "intended_use": "Treatment of Leber's congenital amaurosis", "eu_designation_number": "EU/3/07/484", "status": "Positive", "first_published_date": "22/01/2008", "last_updated_date": "09/08/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-484" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "adeno-associated viral vector serotype 5 containing the human RLBP1 gene", "date_of_designation_or_refusal": "14/10/2016", "intended_use": "Treatment of retinitis pigmentosa", "eu_designation_number": "EU/3/16/1741", "status": "Positive", "first_published_date": "15/11/2016", "last_updated_date": "09/08/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1741" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Mazindol", "date_of_designation_or_refusal": "09/10/2015", "intended_use": "Treatment of narcolepsy", "eu_designation_number": "EU/3/15/1547", "status": "Positive", "first_published_date": "16/11/2015", "last_updated_date": "03/08/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1547" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "bilayer engineered collagen hydrogel-based skin graft composed of autologous keratinocytes and fibroblasts", "date_of_designation_or_refusal": "14/12/2015", "intended_use": "Treatment of partial deep dermal and full-thickness burns", "eu_designation_number": "EU/3/15/1596", "status": "Positive", 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"date_of_designation_or_refusal": "18/07/2022", "intended_use": "Treatment of diffuse large B-cell lymphoma", "eu_designation_number": "EU/3/15/1494", "status": "Withdrawn", "first_published_date": "30/06/2015", "last_updated_date": "19/07/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1494" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "norucholic acid", "date_of_designation_or_refusal": "13/04/2022", "intended_use": "Treatment of primary biliary cholangitis", "eu_designation_number": "EU/3/22/2597", "status": "Positive", "first_published_date": "19/07/2022", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2597" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "sirolimus", "date_of_designation_or_refusal": "20/06/2017", "intended_use": "Treatment of tuberous sclerosis", "eu_designation_number": "EU/3/17/1886", "status": "Positive", "first_published_date": "17/07/2017", "last_updated_date": "19/07/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1886" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "adeno-associated virus serotype 9 containing human MYBPC3 gene", "date_of_designation_or_refusal": "13/04/2022", "intended_use": "Treatment of Hypertrophic cardiomyopathy due to mutations in the MYBPC3 gene encoding cardiac myosin-binding protein C", "eu_designation_number": "EU/3/22/2596", "status": "Positive", "first_published_date": "18/07/2022", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2596" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Glofitamab", "date_of_designation_or_refusal": "13/04/2022", "intended_use": "Treatment of mantle cell lymphoma", "eu_designation_number": "EU/3/22/2595", "status": "Positive", "first_published_date": "18/07/2022", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2595" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "autologous CD34+ haematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LV, encoding for the alpha-L-iduronidase cDNA", "date_of_designation_or_refusal": "26/10/2018", "intended_use": "Treatment of mucopolysaccharidosis type I", "eu_designation_number": "EU/3/18/2073", "status": "Positive", "first_published_date": "10/01/2019", "last_updated_date": "14/07/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2073" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene", "date_of_designation_or_refusal": "10/06/2014", "intended_use": "Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome)", "eu_designation_number": "EU/3/14/1280", "status": "Positive", "first_published_date": "09/07/2014", "last_updated_date": "14/07/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1280" }, { "medicine_name": "Libmeldy", "related_ema_product_number": "EMEA/H/C/005321", "active_substance": "autologous CD34+ cells transfected with lentiviral vector containing the human arylsulfatase A cDNA", "date_of_designation_or_refusal": "13/04/2007", "intended_use": "treatment of metachromatic leukodystrophy", "eu_designation_number": "EU/3/07/446", "status": "Positive", "first_published_date": "29/07/2008", "last_updated_date": "14/07/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-446" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Darinaparsin", "date_of_designation_or_refusal": "15/04/2011", "intended_use": "Treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic/disseminated)", "eu_designation_number": "EU/3/11/850", "status": "Positive", "first_published_date": "26/04/2011", "last_updated_date": "14/07/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-850" }, { "medicine_name": "Yescarta", "related_ema_product_number": "EMEA/H/C/004480", "active_substance": "autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (axicabtagene ciloleucel)", "date_of_designation_or_refusal": "11/11/2015", "intended_use": "Treatment of follicular lymphoma", "eu_designation_number": "EU/3/15/1579", "status": "Positive", "first_published_date": "06/01/2016", "last_updated_date": "12/07/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1579" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "adeno-associated virus serotype 9 vector containing human n-acetylgalactosamine-6-sulfate sulfatase gene", "date_of_designation_or_refusal": "09/01/2020", "intended_use": "Treatment of mucopolysaccharidosis type IVA (Morquio A Syndrome)", "eu_designation_number": "EU/3/19/2238", "status": "Positive", "first_published_date": "19/05/2020", "last_updated_date": "08/07/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2238" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "adeno-associated viral vector serotype 9 containing the human sulfamidase gene", "date_of_designation_or_refusal": "21/06/2011", "intended_use": "Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome)", "eu_designation_number": "EU/3/11/877", "status": "Expired", "first_published_date": "12/07/2011", "last_updated_date": "08/07/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-877" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "recombinant human monoclonal antibody binding to vascular adhesion protein-1", "date_of_designation_or_refusal": "19/03/2015", "intended_use": "Treatment of primary sclerosing cholangitis", "eu_designation_number": "EU/3/15/1461", "status": "Positive", "first_published_date": "11/05/2015", "last_updated_date": "08/07/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1461" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "N-Hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide", "date_of_designation_or_refusal": "26/04/2012", "intended_use": "Treatment of neurofibromatosis type 2", "eu_designation_number": "EU/3/12/993", "status": "Positive", "first_published_date": "18/06/2012", "last_updated_date": "05/07/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-993" }, { "medicine_name": "Vynpenta (previously AvacopanChemoCentryx); Tavneos", "related_ema_product_number": "EMEA/H/C/004487; EMEA/H/C/005523", "active_substance": "(2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amNide", "date_of_designation_or_refusal": "19/11/2014", "intended_use": "Treatment of microscopic polyangiitis (avacopan)", "eu_designation_number": "EU/3/14/1372", "status": "Positive", "first_published_date": "15/01/2015", "last_updated_date": "04/07/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1372" }, { "medicine_name": "Vynpenta (previously AvacopanChemoCentryx); Tavneos", "related_ema_product_number": "EMEA/H/C/004487; EMEA/H/C/005523", "active_substance": "", "date_of_designation_or_refusal": "19/11/2014", "intended_use": "Treatment of granulomatosis with polyangiitis", "eu_designation_number": "EU/3/14/1373", "status": "Positive", "first_published_date": "15/01/2015", "last_updated_date": "04/07/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1373" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "human autologous mesenchymal adult stem cells extracted from adipose tissue", "date_of_designation_or_refusal": "26/08/2005", "intended_use": "Treatment of anal fistula", "eu_designation_number": "EU/3/05/303", "status": "Withdrawn", "first_published_date": "22/10/2009", "last_updated_date": "01/07/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-05-303" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "setmelanotide", "date_of_designation_or_refusal": "09/01/2020", "intended_use": "Treatment of Alström syndrome", "eu_designation_number": "EU/3/19/2245", "status": "Positive", "first_published_date": "20/05/2020", "last_updated_date": "01/07/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2245" }, { "medicine_name": "Imcivree", "related_ema_product_number": "EMEA/H/C/005089", "active_substance": "setmelanotide", "date_of_designation_or_refusal": "19/11/2018", "intended_use": "Treatment of leptin receptor deficiency", "eu_designation_number": "EU/3/18/2101", "status": "Positive", "first_published_date": "25/02/2019", "last_updated_date": "01/07/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2101" }, { "medicine_name": "Imcivree", "related_ema_product_number": "EMEA/H/C/005089", "active_substance": "setmelanotide", "date_of_designation_or_refusal": "14/07/2016", "intended_use": "Treatment of pro-opiomelanocortin deficiency", "eu_designation_number": "EU/3/16/1703", "status": "Positive", "first_published_date": "06/09/2016", "last_updated_date": "01/07/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1703" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "setmelanotide", "date_of_designation_or_refusal": "27/06/2016", "intended_use": "Treatment of Prader-Willi syndrome", "eu_designation_number": "EU/3/16/1688", "status": "Positive", "first_published_date": "26/07/2016", "last_updated_date": "01/07/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1688" }, { "medicine_name": "Rezzayo", "related_ema_product_number": "EMEA/H/C/005900", "active_substance": "rezafungin acetate", "date_of_designation_or_refusal": "06/01/2021", "intended_use": "Treatment of invasive candidiasis", "eu_designation_number": "EU/3/20/2385", "status": "Positive", "first_published_date": "18/05/2021", "last_updated_date": "30/06/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2385" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "recombinant human monoclonal antibody to insulin receptor", "date_of_designation_or_refusal": "14/07/2016", "intended_use": "Treatment of congenital hyperinsulinism", "eu_designation_number": "EU/3/16/1702", "status": "Positive", "first_published_date": "06/09/2016", "last_updated_date": "30/06/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1702" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "2-methyl-1-[(4-[6-(trifluoromethyl)pyridin-2-yl]-6-{[2-(trifluoromethyl)pyridin-4-yl]amino}-1,3,5-triazin-2-yl)amino]propan-2-ol methanesulfonate (enasidenib)", "date_of_designation_or_refusal": "28/04/2016", "intended_use": "Treatment of acute myeloid leukaemia", "eu_designation_number": "EU/3/16/1640", "status": "Positive", "first_published_date": "31/05/2016", "last_updated_date": "29/06/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1640" }, { "medicine_name": "Lunsumio", "related_ema_product_number": "EMEA/H/C/005680", "active_substance": "mosunetuzumab", "date_of_designation_or_refusal": "12/11/2021", "intended_use": "Treatment of follicular lymphoma", "eu_designation_number": "EU/3/21/2517", "status": "Positive", "first_published_date": "08/06/2022", "last_updated_date": "23/06/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2517" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "chimeric locked nucleic acid deoxynucleoside phosphorothioate-linked oligonucleotide inhibitor directed against microRNA-155-5p", "date_of_designation_or_refusal": "22/05/2017", "intended_use": "Treatment of cutaneous T-cell lymphoma", "eu_designation_number": "EU/3/17/1872", "status": "Withdrawn", "first_published_date": "29/06/2017", "last_updated_date": "22/06/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1872" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "protein-based delivery vector carrying a DNA payload encoding an RNA-guided nuclease that targets stx genes of Shiga toxin-producing Escherichia coli", "date_of_designation_or_refusal": "21/08/2020", "intended_use": "Prevention of haemolytic uraemic syndrome", "eu_designation_number": "EU/3/20/2316", "status": "Positive", "first_published_date": "25/11/2020", "last_updated_date": "22/06/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2316" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "modified messenger 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"related_ema_product_number": "", "active_substance": "heterologous human adult liver-derived stem cells", "date_of_designation_or_refusal": "16/03/2022", "intended_use": "Treatment of argininosuccinic aciduria", "eu_designation_number": "EU/3/22/2594", "status": "Positive", "first_published_date": "20/06/2022", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2594" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "emactuzumab", "date_of_designation_or_refusal": "16/03/2022", "intended_use": "Treatment of tenosynovial giant cell tumour, localised and diffuse type", "eu_designation_number": "EU/3/22/2591", "status": "Positive", "first_published_date": "20/06/2022", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2591" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "", "date_of_designation_or_refusal": "14/01/2022", "intended_use": "Treatment of Leber’s hereditary optic neuropathy", "eu_designation_number": "EU/3/21/2571", "status": "Positive", "first_published_date": "16/06/2022", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2571" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "(S)-12-fluoro-4-(2-methylpyridin-3-yl)-7a,8,13,14-tetrahydro-7H-[1,2,4]triazolo[4',3':1,6]pyrido[3,2-b]benzofuro[4,3-fg][1,4]oxazonine", "date_of_designation_or_refusal": "16/03/2022", "intended_use": "Treatment of sickle cell disease", "eu_designation_number": "EU/3/22/2589", "status": "Positive", "first_published_date": "16/06/2022", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-22-2589" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "tropatepine 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"2’-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-P-thioguanylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-P-thioadenylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-P-thioadenylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-P-thioadenylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-P-thioguanoylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-P-thioadenylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-P-thioguanylyl-(3’-O->5’-O)-2’-O-(2-methoxyethyl)-P-thioguanylyl ...", "date_of_designation_or_refusal": "10/12/2021", "intended_use": "Treatment of cystic fibrosis", "eu_designation_number": "EU/3/21/2552", "status": "Positive", "first_published_date": "05/05/2022", "last_updated_date": "", "orphan_designation_url": 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biliary tract cancer", "eu_designation_number": "EU/3/18/2112", "status": "Withdrawn", "first_published_date": "11/03/2019", "last_updated_date": "07/02/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2112" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "recombinant anti-CD3-bi-single-chain-Fv-diphtheria toxin fusion protein", "date_of_designation_or_refusal": "09/08/2012", "intended_use": "Treatment of cutaneous T-cell lymphoma", "eu_designation_number": "EU/3/12/1039", "status": "Positive", "first_published_date": "27/09/2012", "last_updated_date": "04/02/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1039" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "recombinant anti-CD3-bi-single-chain-Fv-diphtheria toxin fusion protein", "date_of_designation_or_refusal": "09/08/2012", "intended_use": "Treatment 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"02/08/2007", "intended_use": "Treatment of orthostatic hypotension in patients with pure autonomic failure", "eu_designation_number": "EU/3/07/465", "status": "Withdrawn", "first_published_date": "21/09/2009", "last_updated_date": "28/01/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-465" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "chimeric fibril-reactive IgG1k monoclonal antibody 11-1F4", "date_of_designation_or_refusal": "13/11/2019", "intended_use": "Treatment of AL amyloidosis", "eu_designation_number": "EU/3/19/2222", "status": "Positive", "first_published_date": "13/02/2020", "last_updated_date": "28/01/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2222" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "nomacopan", "date_of_designation_or_refusal": "26/06/2020", "intended_use": "Treatment of 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"27/06/2016", "intended_use": "Treatment of Guillain-Barré syndrome", "eu_designation_number": "EU/3/16/1687", "status": "Positive", "first_published_date": "26/07/2016", "last_updated_date": "26/01/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1687" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "recombinant derivative of C3 transferase", "date_of_designation_or_refusal": "05/09/2008", "intended_use": "Treatment of traumatic spinal cord injury", "eu_designation_number": "EU/3/08/563", "status": "Positive", "first_published_date": "24/04/2009", "last_updated_date": "25/01/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-563" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "1-(2,2-difluoro-1,3-benzodioxol-5-yl)-N-{1-[(2R)-2,3-dihydroxypropyl]-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl}cyclopropanecarboxamide (tezacaftor)", "date_of_designation_or_refusal": "04/07/2014", "intended_use": "Treatment of cystic fibrosis", "eu_designation_number": "EU/3/14/1281", "status": "Positive", "first_published_date": "15/07/2014", "last_updated_date": "25/01/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1281" }, { "medicine_name": "Symkevi", "related_ema_product_number": "EMEA/H/C/004682", "active_substance": "ivacaftor;tezacaftor", "date_of_designation_or_refusal": "27/02/2017", "intended_use": "Treatment of cystic fibrosis", "eu_designation_number": "EU/3/17/1828", "status": "Positive", "first_published_date": "31/03/2017", "last_updated_date": "25/01/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1828" }, { "medicine_name": 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"date_of_designation_or_refusal": "13/04/2021", "intended_use": "Treatment of multiple myeloma", "eu_designation_number": "EU/3/21/2424", "status": "Positive", "first_published_date": "24/01/2022", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2424" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "psilocybine", "date_of_designation_or_refusal": "15/10/2021", "intended_use": "Treatment of fragile X syndrome", "eu_designation_number": "EU/3/21/2513", "status": "Positive", "first_published_date": "21/01/2022", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2513" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Idursulfase beta", "date_of_designation_or_refusal": "15/10/2021", "intended_use": "Treatment of mucopolysaccharidosis type II (Hunter's syndrome)", "eu_designation_number": "EU/3/21/2512", "status": "Positive", "first_published_date": "21/01/2022", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2512" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "allogeneic umbilical cord mesenchymal cells-derived extracellular vesicles", "date_of_designation_or_refusal": "15/10/2021", "intended_use": "Prevention of bronchopulmonary dysplasia", "eu_designation_number": "EU/3/21/2508", "status": "Positive", "first_published_date": "21/01/2022", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2508" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "adeno-associated viral vector serotype 5 expressing the human Cone-Rod Homeobox gene", "date_of_designation_or_refusal": "15/10/2021", "intended_use": "Treatment of cone-rod dystrophy", 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"", "active_substance": "adeno-associated virus serotype hu68 containing the human GALC gene", "date_of_designation_or_refusal": "26/03/2021", "intended_use": "Treatment of Krabbe disease", "eu_designation_number": "EU/3/21/2417", "status": "Positive", "first_published_date": "19/01/2022", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2417" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Fenretinide", "date_of_designation_or_refusal": "14/10/2016", "intended_use": "Treatment of peripheral T-cell lymphoma", "eu_designation_number": "EU/3/16/1751", "status": "Withdrawn", "first_published_date": "15/11/2016", "last_updated_date": "19/01/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1751" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Fenretinide", "date_of_designation_or_refusal": "21/03/2016", "intended_use": "Treatment of cutaneous T-cell lymphoma", "eu_designation_number": "EU/3/16/1630", "status": "Withdrawn", "first_published_date": "03/05/2016", "last_updated_date": "19/01/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1630" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "chimeric fusion protein of recombinant human alpha-N-acetylglucosaminidase and human insulin-like growth factor 2 (tralesinidase alfa)", "date_of_designation_or_refusal": "15/01/2015", "intended_use": "Treatment of mucopolysaccharidosis type IIIB", "eu_designation_number": "EU/3/14/1422", "status": "Positive", "first_published_date": "06/03/2015", "last_updated_date": "14/01/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1422" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "resiquimod", "date_of_designation_or_refusal": "28/04/2016", "intended_use": "Treatment of cutaneous T-cell lymphoma", "eu_designation_number": "EU/3/16/1653", "status": "Positive", "first_published_date": "31/05/2016", "last_updated_date": "13/01/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1653" }, { "medicine_name": "Orepaxam", "related_ema_product_number": "EMEA/H/C/005990", "active_substance": "treprostinil diethanolamine", "date_of_designation_or_refusal": "26/08/2005", "intended_use": "Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension", "eu_designation_number": "EU/3/05/310", "status": "Positive", "first_published_date": "23/09/2009", "last_updated_date": "13/01/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-05-310" }, { "medicine_name": "Artesunate Amivas", "related_ema_product_number": "EMEA/H/C/005550", "active_substance": "artesunate", "date_of_designation_or_refusal": "28/02/2020", "intended_use": "Treatment of malaria", "eu_designation_number": "EU/3/20/2251", "status": "Positive", "first_published_date": "06/05/2020", "last_updated_date": "13/01/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2251" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (axicabtagene ciloleucel)", "date_of_designation_or_refusal": "09/10/2015", "intended_use": "Treatment of mantle cell lymphoma", "eu_designation_number": "EU/3/15/1552", "status": "Withdrawn", "first_published_date": "16/11/2015", "last_updated_date": "12/01/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1552" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (axicabtagene ciloleucel)", "date_of_designation_or_refusal": "11/11/2015", "intended_use": "Treatment of acute lymphoblastic leukaemia", "eu_designation_number": "EU/3/15/1571", "status": "Withdrawn", "first_published_date": "06/01/2016", "last_updated_date": "12/01/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1571" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (axicabtagene ciloleucel)", "date_of_designation_or_refusal": "11/11/2015", "intended_use": "Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma", "eu_designation_number": "EU/3/15/1572", "status": "Withdrawn", "first_published_date": "06/01/2016", "last_updated_date": "12/01/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1572" }, { "medicine_name": "Filspari", "related_ema_product_number": "EMEA/H/C/005783", "active_substance": "sparsentan", "date_of_designation_or_refusal": "19/10/2020", "intended_use": "Treatment of primary IgA nephropathy", "eu_designation_number": "EU/3/20/2345", "status": "Positive", "first_published_date": "16/02/2021", "last_updated_date": "12/01/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2345" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "4'-[(2-butyl-4-oxo-1,3-diazaspiro[4.4]non-1-en-3-yl)methyl]-N-(4,5-dimethyl-3-isoxazolyl)-2'-(ethoxymethyl)-[1,1'-biphenyl]-2-sulfonamide", "date_of_designation_or_refusal": "11/11/2015", "intended_use": "Treatment of focal segmental glomerulosclerosis", "eu_designation_number": "EU/3/15/1574", "status": "Positive", "first_published_date": "06/01/2016", "last_updated_date": "12/01/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1574" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Human plasminogen", "date_of_designation_or_refusal": "28/07/2015", "intended_use": "Treatment of plasminogen deficiency", "eu_designation_number": "EU/3/15/1511", "status": "Positive", "first_published_date": "18/08/2015", "last_updated_date": "06/01/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1511" }, { "medicine_name": "Albrioza", "related_ema_product_number": "EMEA/H/C/005901", "active_substance": "sodium phenylbutyrate;tauroursodeoxycholic acid", "date_of_designation_or_refusal": "04/06/2020", "intended_use": "Treatment of amyotrophic lateral sclerosis", "eu_designation_number": "EU/3/20/2284", "status": "Positive", "first_published_date": "19/08/2020", "last_updated_date": "05/01/2022", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2284" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "sirolimus", "date_of_designation_or_refusal": "19/07/2021", "intended_use": "Treatment of perivascular epithelioid cell tumours", "eu_designation_number": "EU/3/21/2467", "status": "Positive", "first_published_date": "12/11/2021", "last_updated_date": "21/12/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2467" }, { "medicine_name": "Aspaveli", "related_ema_product_number": "EMEA/H/C/005553", "active_substance": "Poly(oxy-1,2-ethanediyl),.alpha.-hydro-.omega.-hydroxy-,15,15'-diester with N-acetyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-.alpha.-aspartyl-L-tryptophylglycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-L-threonyl-2-[2-(2-aminoethoxy)ethoxy]acetyl-N6-carboxy-L-lysinamide cyclic (2.fwdarw.12)-(disulfide); where two identical synthetic peptide domains are covalently linked at the ends of the polyethylene glycol chain", "date_of_designation_or_refusal": "22/05/2017", "intended_use": "Treatment of paroxysmal nocturnal haemoglobinuria", "eu_designation_number": "EU/3/17/1873", "status": "Positive", "first_published_date": "29/06/2017", "last_updated_date": "16/12/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1873" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "zanubrutinib", "date_of_designation_or_refusal": "29/05/2019", "intended_use": "Treatment of lymphoplasmacytic lymphoma", "eu_designation_number": "EU/3/19/2167", "status": "Withdrawn", "first_published_date": "12/08/2019", "last_updated_date": "15/12/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2167" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "tislelizumab", "date_of_designation_or_refusal": "13/11/2020", "intended_use": "Treatment of oesophageal cancer", "eu_designation_number": "EU/3/20/2357", "status": "Withdrawn", "first_published_date": "15/02/2021", "last_updated_date": "14/12/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2357" }, { "medicine_name": "Folcepri", "related_ema_product_number": "EMEA/H/C/002570", "active_substance": "N-[4-[[(2-amino-3,4-dihydro-4-oxo-6-pteridinyl)methyl]amino]benzoyl]-D-gamma-glutamyl-(2S)-2-amino-beta-alanyl-L-alpha-aspartyl-L-cysteine (etarfolatide)", "date_of_designation_or_refusal": "10/09/2012", "intended_use": "Diagnosis of positive folate-receptor status in ovarian cancer", "eu_designation_number": "EU/3/12/1043", "status": "Withdrawn", "first_published_date": "27/09/2012", "last_updated_date": "07/12/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1043" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human beta A-T87Q-globin gene", "date_of_designation_or_refusal": "29/04/2014", "intended_use": "The treatment of sickle cell disease", "eu_designation_number": "EU/3/14/1263", "status": "Withdrawn", "first_published_date": "04/06/2014", "last_updated_date": "07/12/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1263" }, { "medicine_name": "Abecma", "related_ema_product_number": "EMEA/H/C/004662", "active_substance": "autologous T lymphocyte-enriched population of cells transduced with a lentiviral vector encoding a chimeric antigen receptor targeting human B cell maturation antigen with 4-1BB and CD3-zeta intracellular signalling domains (idecabtagene vicleucel)", "date_of_designation_or_refusal": "20/04/2017", "intended_use": "Treatment of multiple myeloma", "eu_designation_number": "EU/3/17/1863", "status": "Positive", "first_published_date": "15/05/2017", "last_updated_date": "03/12/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1863" }, { "medicine_name": "Livmarli", "related_ema_product_number": "EMEA/H/C/005551", "active_substance": "(4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride (maralixibat chloride)", "date_of_designation_or_refusal": "16/01/2014", "intended_use": "Treatment of progressive familial intrahepatic cholestasis", "eu_designation_number": "EU/3/13/1216", "status": "Positive", "first_published_date": "22/01/2014", "last_updated_date": "03/12/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1216" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Glycosylation independent lysosomal targeting (GILT)-tagged recombinant human acid alpha glucosidase", "date_of_designation_or_refusal": "27/10/2011", "intended_use": "Treatment of glycogen storage disease type II (Pompe's disease)", "eu_designation_number": "EU/3/11/921", "status": "Positive", "first_published_date": "23/11/2011", "last_updated_date": "30/11/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-921" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "CD34+ haematopoietic stem and progenitor cells with CD3+ T-cells", "date_of_designation_or_refusal": "24/08/2018", "intended_use": "Treatment in solid organ transplantation", "eu_designation_number": "EU/3/18/2063", "status": "Positive", "first_published_date": "20/12/2018", "last_updated_date": "30/11/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2063" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate (tafamidis)", "date_of_designation_or_refusal": "28/08/2006", "intended_use": "Treatment of familial amyloid polyneuropathy", "eu_designation_number": "EU/3/06/401", "status": "Expired", "first_published_date": "02/04/2009", "last_updated_date": "25/11/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-06-401" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "hydrocortisone", "date_of_designation_or_refusal": "22/05/2006", "intended_use": "Treatment of adrenal insufficiency", "eu_designation_number": "EU/3/06/372", "status": "Expired", "first_published_date": "21/09/2009", "last_updated_date": "25/11/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-06-372" }, { "medicine_name": "Qinlock", "related_ema_product_number": "EMEA/H/C/005614", "active_substance": "1-[4-bromo-5-[1-ethyl-7-(methylamino)-2-oxo-1,2-dihydro-1,6-naphthyridin-3-yl]-2-fluorophenyl]-3-phenylurea", "date_of_designation_or_refusal": "08/11/2017", "intended_use": "Treatment of gastrointestinal stromal tumours", "eu_designation_number": "EU/3/17/1936", "status": "Positive", "first_published_date": "15/01/2018", "last_updated_date": "23/11/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1936" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Ibrutinib", "date_of_designation_or_refusal": "16/01/2014", "intended_use": "Treatment of follicular lymphoma", "eu_designation_number": "EU/3/13/1212", "status": "Withdrawn", "first_published_date": "23/01/2014", "last_updated_date": "23/11/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1212" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Ibrutinib", "date_of_designation_or_refusal": "13/11/2013", "intended_use": "Treatment of diffuse large B-cell lymphoma", "eu_designation_number": "EU/3/13/1203", "status": "Withdrawn", "first_published_date": "02/12/2013", "last_updated_date": "23/11/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1203" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Ibrutinib", "date_of_designation_or_refusal": "18/11/2016", "intended_use": "Treatment of graft-versus-host disease", "eu_designation_number": "EU/3/16/1780", "status": "Withdrawn", "first_published_date": "14/12/2016", "last_updated_date": "23/11/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1780" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Ibrutinib", "date_of_designation_or_refusal": "10/08/2015", "intended_use": "Treatment of marginal zone lymphoma", "eu_designation_number": "EU/3/15/1541", "status": "Withdrawn", "first_published_date": "02/10/2015", "last_updated_date": "23/11/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1541" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Humanised Fc engineered monoclonal antibody against CD19 (tafasitamab)", "date_of_designation_or_refusal": "04/07/2014", "intended_use": "Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma", "eu_designation_number": "EU/3/14/1286", "status": "Positive", "first_published_date": "16/07/2014", "last_updated_date": "18/11/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1286" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "recombinant human ectonucleotide pyrophosphatase/phosphodiesterase 1 fused to the Fc fragment of IgG1", "date_of_designation_or_refusal": "19/07/2021", "intended_use": "Treatment of adenosine triphosphate binding cassette transporter protein subfamily C member 6 deficiency", "eu_designation_number": "EU/3/21/2479", "status": "Positive", "first_published_date": "16/11/2021", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2479" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "mRNA encoding the human glycogen debranching enzyme", "date_of_designation_or_refusal": "19/07/2021", "intended_use": "Treatment of glycogen storage disease type III", "eu_designation_number": "EU/3/21/2472", "status": "Positive", "first_published_date": "16/11/2021", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-21-2472" }, { "medicine_name": "", 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"https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-698" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "carbetocin", "date_of_designation_or_refusal": "21/03/2012", "intended_use": "Treatment of Prader-Willi syndrome", "eu_designation_number": "EU/3/12/975", "status": "Positive", "first_published_date": "19/04/2012", "last_updated_date": "13/10/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-975" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "allogeneic fetal human retinal progenitor cells expanded ex vivo", "date_of_designation_or_refusal": "17/02/2016", "intended_use": "Treatment of retinitis pigmentosa", "eu_designation_number": "EU/3/16/1620", "status": "Positive", "first_published_date": "07/04/2016", "last_updated_date": "13/10/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1620" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "myriocin", "date_of_designation_or_refusal": "12/02/2015", "intended_use": "Treatment of retinitis pigmentosa", "eu_designation_number": "EU/3/15/1449", "status": "Positive", "first_published_date": "09/04/2015", "last_updated_date": "13/10/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1449" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "(E)-(6-((N-methyl-((3-methylbenzofuran-2-yl)methyl)amino)-3-oxoprop-1-en-1-yl)-2-oxo-3,4-dihydro-1,8-naphthyridin-1(2H)-yl)methyl phosphate, bis ethanolamine salt (afabicin)", "date_of_designation_or_refusal": "14/10/2016", "intended_use": "Treatment of osteomyelitis", "eu_designation_number": "EU/3/16/1737", "status": "Positive", "first_published_date": "15/11/2016", "last_updated_date": 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"first_published_date": "23/07/2018", "last_updated_date": "07/10/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2027" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "human monoclonal antibody against activin A", "date_of_designation_or_refusal": "18/11/2016", "intended_use": "Treatment of fibrodysplasia ossificans progressiva", "eu_designation_number": "EU/3/16/1779", "status": "Positive", "first_published_date": "14/12/2016", "last_updated_date": "07/10/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1779" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "single-chain urokinase plasminogen activator", "date_of_designation_or_refusal": "16/12/2014", "intended_use": "Treatment of pleural empyema", "eu_designation_number": "EU/3/14/1383", "status": "Positive", "first_published_date": "26/02/2015", "last_updated_date": "27/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1383" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Maralixibat chloride", "date_of_designation_or_refusal": "27/07/2020", "intended_use": "Treatment of biliary atresia", "eu_designation_number": "EU/3/20/2304", "status": "Positive", "first_published_date": "19/11/2020", "last_updated_date": "24/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2304" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "eptacog alfa (activated)", "date_of_designation_or_refusal": "14/12/2005", "intended_use": "Treatment of diffuse alveolar haemorrhage", "eu_designation_number": "EU/3/05/333", "status": "Withdrawn", "first_published_date": "21/09/2009", "last_updated_date": "21/09/2021", "orphan_designation_url": 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"related_ema_product_number": "", "active_substance": "teriparatide", "date_of_designation_or_refusal": "27/06/2016", "intended_use": "Treatment of hypoparathyroidism", "eu_designation_number": "EU/3/16/1689", "status": "Positive", "first_published_date": "26/07/2016", "last_updated_date": "16/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1689" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "exenatide", "date_of_designation_or_refusal": "21/03/2016", "intended_use": "Treatment of idiopathic intracranial hypertension", "eu_designation_number": "EU/3/16/1629", "status": "Positive", "first_published_date": "02/05/2016", "last_updated_date": "16/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1629" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "sodium valproate fNor", "date_of_designation_or_refusal": "15/01/2015", "intended_use": "Treatment of Wolfram syndrome", "eu_designation_number": "EU/3/14/1428", "status": "Positive", "first_published_date": "06/03/2015", "last_updated_date": "16/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1428" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "adenovirus serotype 5 containing partial E1A deletion and an integrin-binding domain (tasadenoturev)", "date_of_designation_or_refusal": "16/12/2014", "intended_use": "Treatment of glioma", "eu_designation_number": "EU/3/14/1396", "status": "Positive", "first_published_date": "26/02/2015", "last_updated_date": "16/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1396" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "hydroxy-propyl-beta-cyclodextrin (hydroxypropylbetadex)", 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"related_ema_product_number": "", "active_substance": "recombinant factor VIIa modified with three terminal repeats derived from the β chain of human chorionic gonadotropin", "date_of_designation_or_refusal": "22/08/2014", "intended_use": "Treatment of haemophilia B", "eu_designation_number": "EU/3/14/1319", "status": "Positive", "first_published_date": "01/10/2014", "last_updated_date": "16/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1319" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "recombinant factor VIIa modified with three terminal repeats derived from the β chain of human chorionic gonadotropin", "date_of_designation_or_refusal": "22/08/2014", "intended_use": "Treatment of congenital factor VII deficiency", "eu_designation_number": "EU/3/14/1312", "status": "Positive", "first_published_date": "30/09/2014", "last_updated_date": "16/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1312" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "recombinant factor VIIa modified with three terminal repeats derived from the β chain of human chorionic gonadotropin", "date_of_designation_or_refusal": "22/08/2014", "intended_use": "Treatment of haemophilia A", "eu_designation_number": "EU/3/14/1316", "status": "Positive", "first_published_date": "01/10/2014", "last_updated_date": "16/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1316" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "axicabtagene ciloleucel", "date_of_designation_or_refusal": "26/06/2020", "intended_use": "Treatment of marginal zone lymphoma", "eu_designation_number": "EU/3/20/2296", "status": "Positive", "first_published_date": "24/09/2020", "last_updated_date": "15/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2296" }, { "medicine_name": "Tecartus", "related_ema_product_number": "EMEA/H/C/005102", "active_substance": "autologous peripheral blood T cells CD4 and CD8 selected and CD3 and CD28 activated transduced with retroviral vector expressing anti CD19 CD28/CD3-zeta chimeric antigen receptor and cultured (brexucabtagene autoleucel)", "date_of_designation_or_refusal": "13/11/2019", "intended_use": "Treatment of mantle cell lymphoma", "eu_designation_number": "EU/3/19/2220", "status": "Positive", "first_published_date": "13/02/2020", "last_updated_date": "15/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2220" }, { "medicine_name": "Yescarta", "related_ema_product_number": "EMEA/H/C/004480", "active_substance": "autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (axicabtagene ciloleucel)", "date_of_designation_or_refusal": "09/10/2015", "intended_use": "Treatment of primary mediastinal large B-cell lymphoma", "eu_designation_number": "EU/3/15/1553", "status": "Positive", "first_published_date": "16/11/2015", "last_updated_date": "15/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1553" }, { "medicine_name": "Koselugo", "related_ema_product_number": "EMEA/H/C/005244", "active_substance": "selumetinib", "date_of_designation_or_refusal": "31/07/2018", "intended_use": "Treatment of neurofibromatosis type 1", "eu_designation_number": "EU/3/18/2050", "status": "Positive", "first_published_date": "11/10/2018", "last_updated_date": "15/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-2050" }, { "medicine_name": "Voxzogo", "related_ema_product_number": "EMEA/H/C/005475", "active_substance": "modified recombinant human C-type natriuretic peptide (Vosoritide)", "date_of_designation_or_refusal": "24/01/2013", "intended_use": "Treatment of achondroplasia", "eu_designation_number": "EU/3/12/1094", "status": "Positive", "first_published_date": "01/03/2013", "last_updated_date": "09/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1094" }, { "medicine_name": "Minjuvi", "related_ema_product_number": "EMEA/H/C/005436", "active_substance": "Humanised Fc engineered monoclonal antibody against CD19 (tafasitamab)", "date_of_designation_or_refusal": "15/01/2015", "intended_use": "Treatment of diffuse large B-cell lymphoma", "eu_designation_number": "EU/3/14/1424", "status": "Positive", "first_published_date": "06/03/2015", "last_updated_date": "08/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1424" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "recombinant human heparan-N-sulfatase", "date_of_designation_or_refusal": "07/11/2008", "intended_use": "Treatment of mucopolysaccharidosis, type IIIA (Sanfilippo A syndrome)", "eu_designation_number": "EU/3/08/582", "status": "Withdrawn", "first_published_date": "29/06/2009", "last_updated_date": "02/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-582" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "chimeric IgG monoclonal antibody cG250 (girentuximab)", "date_of_designation_or_refusal": "19/03/2002", "intended_use": "Treatment of renal-cell carcinoma", "eu_designation_number": "EU/3/02/094", "status": "Withdrawn", "first_published_date": "08/01/2003", "last_updated_date": "02/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-02-094" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Fosbretabulin tromethamine", "date_of_designation_or_refusal": "17/07/2013", "intended_use": "Treatment of ovarian cancer", "eu_designation_number": "EU/3/13/1154", "status": "Withdrawn", "first_published_date": "21/08/2013", "last_updated_date": "02/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1154" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "reduced oxidised N-acetyl heparin (roneparstat)", "date_of_designation_or_refusal": "21/05/2015", "intended_use": "Treatment of plasma cell myeloma", "eu_designation_number": "EU/3/15/1487", "status": "Withdrawn", "first_published_date": "30/06/2015", "last_updated_date": "02/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1487" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "N-(2-((4Z,7Z,10Z,13Z,16Z,19Z)-docosa-4,7,10,13,16,19-hexaenamido)ethyl)-2-hydroxybenzamide (edasalonexent)", "date_of_designation_or_refusal": "09/10/2015", "intended_use": "Treatment of Duchenne muscular dystrophy", "eu_designation_number": "EU/3/15/1560", "status": "Withdrawn", "first_published_date": "18/11/2015", "last_updated_date": "02/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1560" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Fosbretabulin tromethamine", "date_of_designation_or_refusal": "21/03/2016", "intended_use": "Treatment of gastro-entero-pancreatic neuroendocrine tumours", "eu_designation_number": "EU/3/16/1633", "status": "Withdrawn", "first_published_date": "04/05/2016", "last_updated_date": "02/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1633" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "efgartigimod alfa", "date_of_designation_or_refusal": "16/12/2019", "intended_use": "Treatment of immune thrombocytopenia", "eu_designation_number": "EU/3/19/2230", "status": "Positive", "first_published_date": "25/02/2020", "last_updated_date": "02/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2230" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Enoxacin", "date_of_designation_or_refusal": "19/03/2015", "intended_use": "Treatment of amyotrophic lateral sclerosis", "eu_designation_number": "EU/3/15/1459", "status": "Positive", "first_published_date": "11/05/2015", "last_updated_date": "02/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1459" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "4-hydroxyandrostenedione;serenoa serrulata;alpha lipoic acid", "date_of_designation_or_refusal": "28/04/2016", "intended_use": "Treatment of multiple symmetric lipomatosis", "eu_designation_number": "EU/3/16/1646", "status": "Positive", "first_published_date": "31/05/2016", "last_updated_date": "02/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1646" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Ibutamoren mesilate", "date_of_designation_or_refusal": "20/06/2017", "intended_use": "Treatment of growth hormone deficiency", "eu_designation_number": "EU/3/17/1882", "status": "Positive", "first_published_date": "17/07/2017", "last_updated_date": "02/09/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1882" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "heterologous human adult liver-derived stem cells", "date_of_designation_or_refusal": "05/03/2012", "intended_use": "Treatment of carbamoyl-phosphate synthase-1 deficiency", "eu_designation_number": "EU/3/12/971", "status": "Positive", "first_published_date": "19/04/2012", "last_updated_date": "26/08/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-971" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "heterologous human adult liver-derived stem cells", "date_of_designation_or_refusal": "27/09/2011", "intended_use": "Treatment of ornithine transcarbamylase deficiency", "eu_designation_number": "EU/3/11/904", "status": "Positive", "first_published_date": "17/10/2011", "last_updated_date": "26/08/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-904" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "heterologous human adult liver-derived stem cells", "date_of_designation_or_refusal": "26/04/2012", "intended_use": "Treatment of acute liver failure", "eu_designation_number": "EU/3/12/983", "status": "Positive", "first_published_date": "18/06/2012", "last_updated_date": "26/08/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-983" }, { "medicine_name": "Qarziba (previously Dinutuximab beta EUSA and Dinutuximab beta Apeiron)", "related_ema_product_number": "EMEA/H/C/003918", "active_substance": "chimeric monoclonal antibody against GD2", "date_of_designation_or_refusal": "08/11/2012", "intended_use": "Treatment of neuroblastoma", "eu_designation_number": "EU/3/12/1062", "status": "Positive", "first_published_date": "13/12/2012", "last_updated_date": "26/08/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1062" }, { "medicine_name": "Romvimza", "related_ema_product_number": "EMEA/H/C/006363", "active_substance": "2-(isopropylamino)-3-methyl-5-(6-methyl-5-((2-(1-methyl-1H-pyrazol-4-yl)pyridin-4-yl)oxy)pyridin-2-yl)pyrimidin-4(3H)-one", "date_of_designation_or_refusal": "16/12/2019", "intended_use": "Treatment of tenosynovial giant cell tumour, localised and diffuse type", "eu_designation_number": "EU/3/19/2227", "status": "Positive", "first_published_date": "24/02/2020", "last_updated_date": "25/08/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2227" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "murine anti-CD22 antibody variable region fused to truncated Pseudomonas exotoxin 38 ( Moxetumomab pasudotox)", "date_of_designation_or_refusal": "05/12/2008", "intended_use": "Treatment of hairy cell leukaemia", "eu_designation_number": "EU/3/08/592", "status": "Withdrawn", "first_published_date": "17/09/2009", "last_updated_date": "19/08/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-592" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "1, 1'-[1,4-Phenylenebis 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"related_ema_product_number": "", "active_substance": "(RS)-Bacoflen;naltrexone hydrochloride;D-sorbitol", "date_of_designation_or_refusal": "26/03/2014", "intended_use": "Treatment of Charcot-Marie-Tooth disease type 1A", "eu_designation_number": "EU/3/14/1260", "status": "Positive", "first_published_date": "07/05/2014", "last_updated_date": "16/07/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1260" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "human monoclonal antibody targeting Staphylococcus aureus alpha-toxin (tosatoxumab)", "date_of_designation_or_refusal": "05/03/2012", "intended_use": "Treatment of pneumonia caused by Staphylococcus aureus", "eu_designation_number": "EU/3/12/968", "status": "Positive", "first_published_date": "06/07/2012", "last_updated_date": "13/07/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-968" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "gallium citrate", "date_of_designation_or_refusal": "21/08/2019", "intended_use": "Treatment of cystic fibrosis", "eu_designation_number": "EU/3/19/2189", "status": "Positive", "first_published_date": "14/01/2020", "last_updated_date": "13/07/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2189" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "human monoclonal antibody against Pseudomonas aeruginosa serotype O11 (Panobacumab)", "date_of_designation_or_refusal": "29/06/2006", "intended_use": "Treatment of pneumonia caused by serotype O11 Pseudomonas aeruginosa", "eu_designation_number": "EU/3/06/381", "status": "Positive", "first_published_date": "15/05/2009", "last_updated_date": "13/07/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-06-381" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "dimethyl fumarate", "date_of_designation_or_refusal": "21/03/2018", "intended_use": "Treatment of Friedreich’s ataxia", "eu_designation_number": "EU/3/18/1990", "status": "Positive", "first_published_date": "15/05/2018", "last_updated_date": "08/07/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-1990" }, { "medicine_name": "Hemgenix", "related_ema_product_number": "EMEA/H/C/004827", "active_substance": "recombinant adeno-associated viral vector containing a codon-optimized Padua derivative of human coagulation factor IX cDNA (etranacogene dezaparvovec)", "date_of_designation_or_refusal": "21/03/2018", "intended_use": "Treatment of haemophilia B", "eu_designation_number": "EU/3/18/1999", "status": "Positive", "first_published_date": "17/05/2018", "last_updated_date": "01/07/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-1999" }, { "medicine_name": "Hepcludex", "related_ema_product_number": "EMEA/H/C/004854", "active_substance": "", "date_of_designation_or_refusal": "19/06/2015", "intended_use": "Treatment of hepatitis delta virus infection", "eu_designation_number": "EU/3/15/1500", "status": "Positive", "first_published_date": "27/07/2015", "last_updated_date": "30/06/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1500" }, { "medicine_name": "Enspryng", "related_ema_product_number": "EMEA/H/C/004788", "active_substance": "Humanised anti-IL-6 receptor monoclonal antibody (satralizumab)", "date_of_designation_or_refusal": "27/06/2016", "intended_use": "Treatment of neuromyelitis optica spectrum disorders", "eu_designation_number": "EU/3/16/1680", "status": "Positive", "first_published_date": "26/07/2016", "last_updated_date": "30/06/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1680" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "recombinant human acid ceramidase", "date_of_designation_or_refusal": "10/08/2015", "intended_use": "Treatment of cystic fibrosis", "eu_designation_number": "EU/3/15/1536", "status": "Positive", "first_published_date": "02/10/2015", "last_updated_date": "25/06/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1536" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "recombinant human acid ceramidase", "date_of_designation_or_refusal": "19/02/2014", "intended_use": "Treatment of Farber disease", "eu_designation_number": "EU/3/14/1243", "status": "Positive", "first_published_date": "02/04/2014", "last_updated_date": "25/06/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1243" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "recombinant human aspartylglucosaminidase", "date_of_designation_or_refusal": "15/01/2015", "intended_use": "Treatment of aspartylglucosaminuria", "eu_designation_number": "EU/3/14/1410", "status": "Withdrawn", "first_published_date": "05/03/2015", "last_updated_date": "25/06/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1410" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "chimeric group B adenovirus (11p/3) with deletions in the E3 and E4 regions (enadenotucirev)", "date_of_designation_or_refusal": "12/02/2015", "intended_use": "Treatment of ovarian cancer", "eu_designation_number": "EU/3/15/1434", "status": "Withdrawn", "first_published_date": "08/04/2015", "last_updated_date": "25/06/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1434" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "allogeneic hepatoblastoma cells encapsulated in alginate, ex vivo expanded", "date_of_designation_or_refusal": "21/08/2020", "intended_use": "Treatment of acute liver failure", "eu_designation_number": "EU/3/20/2312", "status": "Positive", "first_published_date": "25/11/2020", "last_updated_date": "24/06/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2312" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "bacterial lipase (burlulipase)", "date_of_designation_or_refusal": "07/11/2005", "intended_use": "Treatment of malabsorption due to exocrine pancreatic enzyme insufficiency", "eu_designation_number": "EU/3/05/323", "status": "Positive", "first_published_date": "09/02/2006", "last_updated_date": "21/06/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-05-323" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "hydrocortisone", "date_of_designation_or_refusal": "27/07/2005", "intended_use": "Treatment of congenital adrenal hyperplasia", "eu_designation_number": "EU/3/05/296", "status": "Positive", "first_published_date": "21/09/2009", "last_updated_date": "14/06/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-05-296" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "adeno-associated viral vector serotype 2/2 containing a gene encoding the channelrhodopsin-2 protein", "date_of_designation_or_refusal": "14/10/2016", "intended_use": "Treatment of retinitis pigmentosa", "eu_designation_number": "EU/3/16/1740", "status": "Withdrawn", "first_published_date": "15/11/2016", "last_updated_date": "11/06/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1740" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "5-(4,6-dimorpholino-1,3,5-triazin-2-yl)-4-(trifluoromethyl)pyridin-2-amine (bimiralisib)", "date_of_designation_or_refusal": "27/02/2017", "intended_use": "Treatment of diffuse large B-cell lymphoma", "eu_designation_number": "EU/3/17/1830", "status": "Positive", "first_published_date": "31/03/2017", "last_updated_date": "10/06/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1830" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "carglumic acid", "date_of_designation_or_refusal": "07/11/2008", "intended_use": "Treatment of propionic acidaemia", "eu_designation_number": "EU/3/08/577", "status": "Expired", "first_published_date": "02/04/2009", "last_updated_date": "09/06/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-577" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "carglumic acid", "date_of_designation_or_refusal": "07/11/2008", "intended_use": "Treatment of isovaleric acidaemia", "eu_designation_number": "EU/3/08/575", "status": "Expired", "first_published_date": "02/04/2009", "last_updated_date": "09/06/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-575" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "carglumic acid", "date_of_designation_or_refusal": "07/11/2008", "intended_use": "Treatment of methylmalonic acidaemia", "eu_designation_number": "EU/3/08/576", "status": "Expired", "first_published_date": "02/04/2009", "last_updated_date": "09/06/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-576" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "selinexor", "date_of_designation_or_refusal": "19/11/2014", "intended_use": "Treatment of plasma 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"(S)-3-(1-(9H-purin-6-ylamino)ethyl)-8-chloro-2-phenylisoquinolin-1(2H)-one", "date_of_designation_or_refusal": "17/07/2013", "intended_use": "Treatment of follicular lymphoma", "eu_designation_number": "EU/3/13/1157", "status": "Positive", "first_published_date": "16/08/2013", "last_updated_date": "31/05/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1157" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "(S)-3-(1-(9H-purin-6-ylamino)ethyl)-8-chloro-2-phenylisoquinolin-1(2H)-one (duvelisib)", "date_of_designation_or_refusal": "26/04/2013", "intended_use": "Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma", "eu_designation_number": "EU/3/13/1125", "status": "Positive", "first_published_date": "14/05/2013", "last_updated_date": "31/05/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1125" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "adeno-associated viral vector expressing lipoprotein lipase (alipogene tiparvovec)", "date_of_designation_or_refusal": "09/03/2004", "intended_use": "Treatment of lipoprotein-lipase deficiency", "eu_designation_number": "EU/3/04/194", "status": "Withdrawn", "first_published_date": "07/03/2007", "last_updated_date": "31/05/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-04-194" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "adeno-associated viral vector serotype 9 encoding human ATP7B", "date_of_designation_or_refusal": "09/12/2020", "intended_use": "Treatment of Wilson's disease", "eu_designation_number": "EU/3/20/2378", "status": "Positive", "first_published_date": "27/05/2021", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2378" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "2’-O-(2-methoxyethyl) modified antisense oligonucleotide targeting glycogen synthase 1 pre‑mRNA", "date_of_designation_or_refusal": "09/12/2020", "intended_use": "Treatment of progressive myoclonic epilepsy type 2 (Lafora disease)", "eu_designation_number": "EU/3/20/2377", "status": "Positive", "first_published_date": "27/05/2021", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2377" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "autologous CD4+ and CD8+ T cells genetically modified with a lentiviral vector encoding a B-cell maturation antigen-specific chimeric antigen receptor", "date_of_designation_or_refusal": "09/12/2020", "intended_use": "Treatment of multiple myeloma", "eu_designation_number": "EU/3/20/2366", "status": "Withdrawn", "first_published_date": "26/05/2021", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2366" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Humanised IgG1 monoclonal antibody against the extracellular domain of receptor tyrosine kinase-like orphan receptor 1 coupled via a proteolytically cleavable maleimidocaproyl-valine-citrulline-para-aminobenzoate linker to monomethyl auristatin E", "date_of_designation_or_refusal": "09/12/2020", "intended_use": "Treatment of mantle cell lymphoma", "eu_designation_number": "EU/3/20/2373", "status": "Positive", "first_published_date": "25/05/2021", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2373" }, { "medicine_name": "Spexotras", "related_ema_product_number": "EMEA/H/C/005886", "active_substance": "trametinib dimethyl sulfoxide", "date_of_designation_or_refusal": "09/12/2020", "intended_use": "Treatment of glioma", 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"related_ema_product_number": "", "active_substance": "allogeneic umbilical cord blood CD34+ cells cultured ex vivo with Notch ligand Delta1 (dilanubicel)", "date_of_designation_or_refusal": "17/01/2018", "intended_use": "Treatment in haematopoietic stem cell transplantation", "eu_designation_number": "EU/3/17/1958", "status": "Withdrawn", "first_published_date": "14/03/2018", "last_updated_date": "22/03/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1958" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Levosimendan", "date_of_designation_or_refusal": "22/02/2018", "intended_use": "Treatment of amyotrophic lateral sclerosis", "eu_designation_number": "EU/3/18/1980", "status": "Withdrawn", "first_published_date": "11/04/2018", "last_updated_date": "19/03/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-1980" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Humanised single-chain monoclonal antibody against CD37 (otlertuzumab)", "date_of_designation_or_refusal": "06/12/2012", "intended_use": "Treatment chronic lymphocytic leukaemia", "eu_designation_number": "EU/3/12/1083", "status": "Withdrawn", "first_published_date": "25/01/2013", "last_updated_date": "19/03/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1083" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "adeno-associated viral vector serotype 8 containing the human UGT1A1 gene (volrubigene ralaparvovec)", "date_of_designation_or_refusal": "22/08/2014", "intended_use": "Treatment of Crigler-Najjar syndrome", "eu_designation_number": "EU/3/14/1321", "status": "Withdrawn", "first_published_date": "01/10/2014", "last_updated_date": "19/03/2021", "orphan_designation_url": 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"Treatment of homocystinuria", "eu_designation_number": "EU/3/20/2348", "status": "Positive", "first_published_date": "18/02/2021", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2348" }, { "medicine_name": "Reblozyl", "related_ema_product_number": "EMEA/H/C/004444", "active_substance": "recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain (luspatercept)", "date_of_designation_or_refusal": "22/08/2014", "intended_use": "Treatment of myelodysplastic syndromes", "eu_designation_number": "EU/3/14/1331", "status": "Positive", "first_published_date": "29/09/2014", "last_updated_date": "18/02/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1331" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Luspatercept", "date_of_designation_or_refusal": "28/02/2020", "intended_use": "Treatment of myelofibrosis", "eu_designation_number": "EU/3/20/2255", "status": "Positive", "first_published_date": "06/05/2020", "last_updated_date": "18/02/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2255" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "adeno-associated virus serotype hu68 containing the human GLB1 gene", "date_of_designation_or_refusal": "19/10/2020", "intended_use": "Treatment of GM1 gangliosidosis", "eu_designation_number": "EU/3/20/2346", "status": "Positive", "first_published_date": "16/02/2021", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2346" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "retifanlimab", "date_of_designation_or_refusal": "19/10/2020", "intended_use": "Treatment of anal 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degeneration", "eu_designation_number": "EU/3/20/2340", "status": "Positive", "first_published_date": "15/02/2021", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2340" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "DNA plasmid encoding human transferrin gene", "date_of_designation_or_refusal": "13/11/2020", "intended_use": "Treatment of retinitis pigmentosa", "eu_designation_number": "EU/3/20/2364", "status": "Positive", "first_published_date": "15/02/2021", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2364" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "decitabine;tetrahydrouridine", "date_of_designation_or_refusal": "19/10/2020", "intended_use": "Treatment of sickle cell disease", "eu_designation_number": "EU/3/20/2338", "status": "Positive", "first_published_date": "15/02/2021", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2338" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "(4-{(2S,4S)-4-ethoxy-1-[(5-methoxy-7-methyl-1H-indol-4-yl)methyl]piperidin-2-yl}benzoic acid-hydrogen chloride(1/1))", "date_of_designation_or_refusal": "19/10/2020", "intended_use": "Treatment of primary IgA nephropathy", "eu_designation_number": "EU/3/20/2336", "status": "Positive", "first_published_date": "15/02/2021", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2336" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "triheptanoin", "date_of_designation_or_refusal": "13/11/2020", "intended_use": "Treatment of carnitine palmitoyltransferase I deficiency", "eu_designation_number": "EU/3/20/2360", "status": "Positive", "first_published_date": "15/02/2021", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2360" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "adeno-associated viral vector serotype 9 expressing codon-optimized human GBA gene", "date_of_designation_or_refusal": "13/11/2020", "intended_use": "Treatment of frontotemporal dementia", "eu_designation_number": "EU/3/20/2359", "status": "Positive", "first_published_date": "15/02/2021", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2359" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "L-pyroglutamyl-L-asparaginyl-L-prolyl-D-tyrosyl-D-tryptophan amide", "date_of_designation_or_refusal": "13/11/2020", "intended_use": "Treatment of amyotrophic lateral sclerosis", "eu_designation_number": "EU/3/20/2358", "status": "Positive", "first_published_date": "15/02/2021", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2358" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "anti-(pancreatic adenocarcinoma upregulated factor) IgG1 humanised monoclonal antibody", "date_of_designation_or_refusal": "13/11/2020", "intended_use": "Treatment of pancreatic cancer", "eu_designation_number": "EU/3/20/2355", "status": "Positive", "first_published_date": "15/02/2021", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2355" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "(R)‑tetrahydrofuran‑3‑yl 4‑(6‑(5‑(4‑ethoxy‑1‑isopropylpiperidin‑4‑yl)pyridin-2-yl)pyrrolo[1,2-b]pyridazin-4-yl)piperazine-1-carboxylate sesquisuccinate", "date_of_designation_or_refusal": "13/11/2020", "intended_use": "Treatment of fibrodysplasia 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"EU/3/08/591", "status": "Withdrawn", "first_published_date": "29/06/2009", "last_updated_date": "10/02/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-591" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "1-(3-methylbutanoyl)-L-aspartyl-L-threonyl-L-histidyl-L-phenylalanyl-L-prolyl-(L-cystinyl-L-isoleucyl-[(N6-(S)-4-carboxy-4-palmitamidobutanoyl)-L-lysinyl]-L-phenylalanyl-L-glutamyl-L-prolyl-L-arginyl-L-serinyl-L-lysinyl-L-glycinyl-L-cystinyl)-L-lysinamide, disulfide, acetate salt", "date_of_designation_or_refusal": "19/10/2020", "intended_use": "Treatment of polycythaemia vera", "eu_designation_number": "EU/3/20/2330", "status": "Positive", "first_published_date": "10/02/2021", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2330" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "human monoclonal antibody against Pseudomonas aeruginosa IATS-O1", "date_of_designation_or_refusal": "28/01/2010", "intended_use": "Treatment of pneumonia caused by serotype O1 Pseudomonas aeruginosa", "eu_designation_number": "EU/3/09/705", "status": "Withdrawn", "first_published_date": "25/02/2010", "last_updated_date": "03/02/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-705" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "RNA, [P-deoxy-P-(dimethylamino)] (2',3'-dideoxy-2',3'-imino-2',3'-seco) (2'a→5') (C-m5U-m5U-A-C-A-G-G-C-m5U-C-C-A-A-m5U-A-G-m5U-G-G-m5U-C-A-G-m5U), 5' [P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylaminophosphonamidate], 3'-[2'a-[N2-acetyl-L-arginyl-6-aminohexanoyl-L-arginyl-L-arginyl-β-alanyl-L-arginyl-L-arginyl-6-aminohexanoyl-L-arginyl-L-arginyl-β-alanyl-L-arginyl-6-aminohexanoyl-β-alanyl], octahydrochloride", "date_of_designation_or_refusal": "02/02/2010", "intended_use": "Treatment of Duchenne muscular dystrophy", "eu_designation_number": "EU/3/09/725", "status": "Withdrawn", "first_published_date": "04/03/2010", "last_updated_date": "03/02/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-725" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "sodium thiosulfate", "date_of_designation_or_refusal": "23/02/2011", "intended_use": "Treatment of calciphylaxis", "eu_designation_number": "EU/3/10/848", "status": "Withdrawn", "first_published_date": "07/03/2011", "last_updated_date": "03/02/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-848" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "adeno-associated viral vector serotype 8 containing the human UGT1A1 gene (volrubigene ralaparvovec)", "date_of_designation_or_refusal": "18/11/2016", "intended_use": "Treatment of Crigler-Najjar syndrome", "eu_designation_number": "EU/3/16/1772", "status": "Withdrawn", "first_published_date": "13/12/2016", "last_updated_date": "03/02/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1772" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "heparin-activated recombinant human fibroblast growth factor 1 (on a biodegradable device made from alpha-calcium sulfate hemihydrate)", "date_of_designation_or_refusal": "27/07/2010", "intended_use": "Treatment of traumatic spinal cord injury", "eu_designation_number": "EU/3/10/754", "status": "Withdrawn", "first_published_date": "05/08/2010", "last_updated_date": "03/02/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-754" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Poly(oxy-1,2-ethanediyl), alpha-hydro-omega-hydroxy-,15,15'-diester with N-acetyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-alpha-aspartyl-L-tryptophylglycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-L-threonyl-2-[2-(2-aminoethoxy)ethoxy]acetyl-N6-carboxy-L-lysinamide cyclic (2.fwdarw.12)-(disulfide); where two identical synthetic peptide domains are covalently linked at the ends of the polyethylene glycol chain (pegcetacoplan)", "date_of_designation_or_refusal": "21/08/2019", "intended_use": "Treatment of C3 glomerulopathy", "eu_designation_number": "EU/3/19/2201", "status": "Positive", "first_published_date": "16/01/2020", "last_updated_date": "03/02/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2201" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "2-isopropyl-3H-naphtho[1,2-d]imidazole-4,5-dione", "date_of_designation_or_refusal": "12/12/2017", "intended_use": "Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes", "eu_designation_number": "EU/3/17/1947", "status": "Positive", "first_published_date": "23/01/2018", "last_updated_date": "03/02/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1947" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "ciclosporin", "date_of_designation_or_refusal": "01/10/2010", "intended_use": "Treatment of moderate and severe closed traumatic brain injury", "eu_designation_number": "EU/3/10/791", "status": "Positive", "first_published_date": "14/10/2010", "last_updated_date": "03/02/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-791" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "2-Hydroxyoleic acid", "date_of_designation_or_refusal": "27/10/2011", "intended_use": "Treatment of glioma", "eu_designation_number": "EU/3/11/916", "status": "Positive", "first_published_date": "23/11/2011", "last_updated_date": "03/02/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-916" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "", "date_of_designation_or_refusal": "09/02/2012", "intended_use": "Treatment of West syndrome", "eu_designation_number": "EU/3/12/953", "status": "Withdrawn", "first_published_date": "01/03/2012", "last_updated_date": "03/02/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-953" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "rovalpituzumab tesirine", "date_of_designation_or_refusal": "30/05/2016", "intended_use": "Treatment of small cell lung cancer", "eu_designation_number": "EU/3/16/1667", "status": "Withdrawn", "first_published_date": "23/06/2016", "last_updated_date": "03/02/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1667" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "oxalobacter formigenes strain HC-1", "date_of_designation_or_refusal": "17/02/2006", "intended_use": "Treatment of primary hyperoxaluria", "eu_designation_number": "EU/3/06/354", "status": "Positive", "first_published_date": "15/05/2009", "last_updated_date": "02/02/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-06-354" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "oxalobacter formigenes strain HC-1", "date_of_designation_or_refusal": "15/10/2014", "intended_use": "Treatment of short bowel syndrome", "eu_designation_number": "EU/3/14/1346", "status": "Positive", "first_published_date": "12/11/2014", "last_updated_date": "02/02/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1346" }, { "medicine_name": "Pemazyre", "related_ema_product_number": "EMEA/H/C/005266", "active_substance": "pemigatinib", "date_of_designation_or_refusal": "17/10/2019", "intended_use": "Treatment of myeloid/lymphoid neoplasms with eosinophilia and rearrangement of PDGFRA, PDGFRB, or FGFR1, or with PCM1-JAK", "eu_designation_number": "EU/3/19/2216", "status": "Positive", "first_published_date": "23/01/2020", "last_updated_date": "01/02/2021", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2216" }, { "medicine_name": "Elzonris", "related_ema_product_number": "EMEA/H/C/005031", "active_substance": "recombinant human interleukin-3 truncated diphtheria toxin fusion protein", "date_of_designation_or_refusal": "11/11/2015", "intended_use": "Treatment of blastic plasmacytoid dendritic cell neoplasm", "eu_designation_number": "EU/3/15/1567", "status": "Positive", 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"(3S)-3-{4-[7-(aminocarbonyl)-2H-indazol-2-yl] phenyl} piperidine tosilate monohydrate salt (niraparib)", "date_of_designation_or_refusal": "04/08/2010", "intended_use": "Treatment of ovarian cancer", "eu_designation_number": "EU/3/10/760", "status": "Positive", "first_published_date": "11/08/2010", "last_updated_date": "06/11/2020", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-760" }, { "medicine_name": "Arikayce liposomal", "related_ema_product_number": "EMEA/H/C/005264", "active_substance": "amikacin sulfate", "date_of_designation_or_refusal": "08/04/2014", "intended_use": "Treatment of nontuberculous mycobacterial lung disease", "eu_designation_number": "EU/3/14/1259", "status": "Positive", "first_published_date": "07/05/2014", "last_updated_date": "03/11/2020", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1259" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "amikacin sulfate", "date_of_designation_or_refusal": "25/07/2006", "intended_use": "Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis", "eu_designation_number": "EU/3/06/387", "status": "Positive", "first_published_date": "02/04/2009", "last_updated_date": "03/11/2020", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-06-387" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "5,7-dichloro-2-((ethylamino)methyl)-8-hydroxy-3-methylquinazolin-4(3H)-one mesilate", "date_of_designation_or_refusal": "16/12/2019", "intended_use": "Treatment of multiple system atrophy", "eu_designation_number": "EU/3/19/2228", "status": "Positive", "first_published_date": "25/02/2020", "last_updated_date": "27/10/2020", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-19-2228" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Humanised monoclonal antibody against myostatin (domagrozumab)", "date_of_designation_or_refusal": "08/02/2013", "intended_use": "Treatment of Duchenne muscular dystrophy", "eu_designation_number": "EU/3/13/1105", "status": "Withdrawn", "first_published_date": "04/03/2013", "last_updated_date": "23/10/2020", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1105" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Mepolizumab", "date_of_designation_or_refusal": "12/03/2013", "intended_use": "Treatment of Churg-Strauss syndrome", "eu_designation_number": "EU/3/13/1116", "status": "Withdrawn", "first_published_date": "05/04/2013", "last_updated_date": "23/10/2020", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1116" }, { "medicine_name": "Bosatria; Bosatria", "related_ema_product_number": "EMEA/H/C/001069; EMEA/H/C/001069", 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"related_ema_product_number": "", "active_substance": "synthetic glucagon analogue modified to contain 7 amino acid substitutions", "date_of_designation_or_refusal": "20/06/2017", "intended_use": "Treatment of congenital hyperinsulinism", "eu_designation_number": "EU/3/17/1887", "status": "Positive", "first_published_date": "17/07/2017", "last_updated_date": "25/09/2020", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1887" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Lys40(NODAGA-68Ga)NH2-exendin-4", "date_of_designation_or_refusal": "26/06/2020", "intended_use": "Diagnosis of insulinoma", "eu_designation_number": "EU/3/20/2295", "status": "Positive", "first_published_date": "24/09/2020", "last_updated_date": "", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-20-2295" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": 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"medicine_name": "", "related_ema_product_number": "", "active_substance": "recombinant humanised anti-human interleukin-1 beta monoclonal antibody", "date_of_designation_or_refusal": "01/10/2010", "intended_use": "Treatment of Behçet's Disease", "eu_designation_number": "EU/3/10/796", "status": "Withdrawn", "first_published_date": "14/10/2010", "last_updated_date": "18/07/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-796" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "ambrisentan", "date_of_designation_or_refusal": "11/04/2005", "intended_use": "Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension", "eu_designation_number": "EU/3/05/273", "status": "Withdrawn", "first_published_date": "29/07/2008", "last_updated_date": "18/07/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-05-273" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "thalidomide", "date_of_designation_or_refusal": "20/11/2001", "intended_use": "Treatment of multiple myeloma", "eu_designation_number": "EU/3/01/067", "status": "Withdrawn", "first_published_date": "29/07/2008", "last_updated_date": "18/07/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-01-067" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "arsenic trioxide", "date_of_designation_or_refusal": "12/12/2016", "intended_use": "Treatment of graft-versus-host disease", "eu_designation_number": "EU/3/16/1797", "status": "Positive", "first_published_date": "10/01/2017", "last_updated_date": "16/07/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1797" }, { "medicine_name": "Verkazia; Vekacia; Vekacia", "related_ema_product_number": "EMEA/H/C/004411; EMEA/H/C/000904; 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"(S)-{8-Fluoro-2-2[4-(3-methoxyphenyl)-1-piperazinyl]-3-[2-methoxy-5-(trifluoromethyl)-phenyl]-3, 4-dihydro-4-quinazolinyl} acetic acid (letermovir)", "date_of_designation_or_refusal": "15/04/2011", "intended_use": "Prevention of cytomegalovirus disease in patients with impaired cell-mediated immunity", "eu_designation_number": "EU/3/11/849", "status": "Positive", "first_published_date": "26/04/2011", "last_updated_date": "28/06/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-849" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Cyclo[L-alanyl-L-seryl-L-isoleucyl-L-prolyl-L-prolyl-L-glutaminyl-L-lysyl-L-tyrosyl-D-prolyl-L-prolyl-(2S)-2-aminodecanoyl-L-alpha-glutamyl-L-threonyl]acetate salt", "date_of_designation_or_refusal": "20/03/2013", "intended_use": "Treatment of congenital alpha-1 antitrypsin deficiency", "eu_designation_number": "EU/3/13/1114", "status": "Positive", "first_published_date": 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"medicine_name": "", "related_ema_product_number": "", "active_substance": "synthetic double-stranded RNA oligonucleotide specific to hydroxyacid oxidase 1 gene", "date_of_designation_or_refusal": "28/07/2015", "intended_use": "Treatment of primary hyperoxaluria type 1", "eu_designation_number": "EU/3/15/1528", "status": "Withdrawn", "first_published_date": "18/08/2015", "last_updated_date": "13/06/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1528" }, { "medicine_name": "Kymriah", "related_ema_product_number": "EMEA/H/C/004090", "active_substance": "autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 (tisagenlecleucel)", "date_of_designation_or_refusal": "29/04/2014", "intended_use": "Treatment of B-lymphoblastic leukaemia/lymphoma", "eu_designation_number": "EU/3/14/1266", "status": "Positive", "first_published_date": "04/06/2014", "last_updated_date": 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"first_published_date": "15/01/2015", "last_updated_date": "13/06/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1353" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "staphylococcus aureus protein A", "date_of_designation_or_refusal": "09/10/2015", "intended_use": "Treatment of immune thrombocytopenia", "eu_designation_number": "EU/3/15/1562", "status": "Positive", "first_published_date": "18/11/2015", "last_updated_date": "12/06/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1562" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Poly[2-[(4-{[1-carboxy-2-(hexadecylcarbamoyl)ethyl]sulfanyl}-2,3-bis({2-[((2S)-2-(2-{[(2R)-2-carbamoyl-(2-{[(2S)-1-ethoxy-3-(3-hydroxy-4oxo-1,4-dihydropyridin-1-yl)-1-oxopropan-2-yl]carbamoyl}ethyl]sulfanyl}-3-{[(2S)-1-ethoxy-3-(3-hydroxy-4-oxo-1,4-dihydropyridin-1-yl)-1-oxopropan-2-yl]carbamoyl}propanamido)-3-(3-hydroxy-4-oxo-1,4-dihydropyridin-1-yl)propanoyl ethyl ester))-methoxy]acetyl}oxy)butyl)sulfanyl]-3-(hexadecylcarbamoyl)propanoic acid]-PEG1500-ester]", "date_of_designation_or_refusal": "16/01/2014", "intended_use": "Treatment of dengue", "eu_designation_number": "EU/3/13/1220", "status": "Positive", "first_published_date": "23/01/2014", "last_updated_date": "12/06/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-13-1220" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "human donor haematopoietic stem and progenitor cells that have been treated ex vivo with the protein transduction domain of the HIV-1 transactivation protein fused to MYC transcription factor", "date_of_designation_or_refusal": "12/01/2017", "intended_use": "Treatment in haematopoietic stem cell transplantation", "eu_designation_number": "EU/3/16/1817", "status": "Positive", "first_published_date": "11/07/2017", "last_updated_date": "12/06/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1817" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "para-aminosalicylic acid", "date_of_designation_or_refusal": "17/12/2010", "intended_use": "Treatment of tuberculosis", "eu_designation_number": "EU/3/10/826", "status": "Positive", "first_published_date": "20/01/2011", "last_updated_date": "08/06/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-826" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "(1S,4R,5R,7S)-3,4-dibenzyl-2-oxo-6,8-dioxa-3-azabyciclo[3.2.1]octane-7-carboxylic acid-L-lysine", "date_of_designation_or_refusal": "16/12/2014", "intended_use": "Treatment of neurotrophic keratitis", "eu_designation_number": "EU/3/14/1400", "status": "Positive", "first_published_date": "26/02/2015", "last_updated_date": "08/06/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-14-1400" }, { "medicine_name": "Onpattro", "related_ema_product_number": "EMEA/H/C/004699", "active_substance": "synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA (also known as patisiran)", "date_of_designation_or_refusal": "06/04/2017", "intended_use": "Treatment of transthyretin-mediated amyloidosis", "eu_designation_number": "EU/3/11/857", "status": "Positive", "first_published_date": "26/04/2011", "last_updated_date": "30/05/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-11-857" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "chimeric 2'-O-(2-methoxyethyl) modified oligonucleotide targeted to huntingtin RNA", "date_of_designation_or_refusal": "19/03/2015", "intended_use": "Treatment of Huntington’s disease", "eu_designation_number": "EU/3/15/1453", "status": "Positive", "first_published_date": "11/05/2015", "last_updated_date": "30/05/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1453" }, { "medicine_name": "Rydapt", "related_ema_product_number": "EMEA/H/C/004095", "active_substance": "Midostaurin", "date_of_designation_or_refusal": "04/08/2010", "intended_use": "Treatment of mastocytosis", "eu_designation_number": "EU/3/10/765", "status": "Positive", "first_published_date": "11/08/2010", "last_updated_date": "29/05/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-10-765" }, { "medicine_name": "Rydapt", "related_ema_product_number": "EMEA/H/C/004095", "active_substance": "Midostaurin", "date_of_designation_or_refusal": "29/07/2004", "intended_use": "Treatment of acute myeloid leukaemia", "eu_designation_number": "EU/3/04/214", "status": "Positive", "first_published_date": "11/10/2005", "last_updated_date": "29/05/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-04-214" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "venetoclax", "date_of_designation_or_refusal": "14/10/2016", "intended_use": "Treatment of multiple myeloma", "eu_designation_number": "EU/3/16/1767", "status": "Withdrawn", "first_published_date": "15/11/2016", "last_updated_date": "29/05/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1767" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "Mazindol", "date_of_designation_or_refusal": "12/02/2015", "intended_use": "Treatment of narcolepsy", "eu_designation_number": "EU/3/15/1444", "status": "Withdrawn", "first_published_date": "09/04/2015", "last_updated_date": "29/05/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1444" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "2-[4-(1-methyl-4-pyridin-4-yl-1H-pyrazol-3-yl)-phenoxymethyl]-quinoline succinic acid", "date_of_designation_or_refusal": "14/07/2016", "intended_use": "Treatment of Huntington’s disease", "eu_designation_number": "EU/3/16/1691", "status": "Withdrawn", "first_published_date": "06/09/2016", "last_updated_date": "29/05/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1691" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "yttrium (90Y) edotreotide", "date_of_designation_or_refusal": "04/08/2008", "intended_use": "Treatment of gastro-entero-pancreatic neuroendocrine tumours", "eu_designation_number": "EU/3/08/589", "status": "Withdrawn", "first_published_date": "20/04/2009", "last_updated_date": "29/05/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-589" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "olaparib", "date_of_designation_or_refusal": "06/12/2007", "intended_use": "Treatment of ovarian cancer", "eu_designation_number": "EU/3/07/501", "status": "Withdrawn", "first_published_date": "02/07/2008", "last_updated_date": "28/05/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-501" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "adeno-associated viral vector serotype 9 encoding miRNA against human superoxide dismutase 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"first_published_date": "03/10/2016", "last_updated_date": "03/05/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-16-1727" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "4-amino-1-[(1S,4R,5S)-2-fluoro-4,5-dihydroxy-3-(hydroxymethyl)cyclopent-2-en-1-yl]pyrimidin-2-one", "date_of_designation_or_refusal": "12/10/2017", "intended_use": "Treatment of pancreatic cancer", "eu_designation_number": "EU/3/17/1937", "status": "Positive", "first_published_date": "15/01/2018", "last_updated_date": "03/05/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-17-1937" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "2'-O-(2-Methoxyethyl) phosphorothioate antisense oligonucleotide targeting the growth hormone receptor", "date_of_designation_or_refusal": "27/06/2016", "intended_use": "Treatment of acromegaly", 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truncated form of the Lm protein listeriolysin O", "date_of_designation_or_refusal": "14/12/2015", "intended_use": "Treatment of osteosarcoma", "eu_designation_number": "EU/3/15/1595", "status": "Positive", "first_published_date": "03/02/2016", "last_updated_date": "03/05/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-15-1595" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "recombinant human monoclonal antibody against mannan-binding lectin-associated serine protease-2", "date_of_designation_or_refusal": "22/02/2018", "intended_use": "Treatment of primary IgA nephropathy", "eu_designation_number": "EU/3/18/1984", "status": "Positive", "first_published_date": "11/04/2018", "last_updated_date": "11/04/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-1984" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "(R)-2-(5-cyano-2-(6-(methoxycarbonyl)-7-methyl-3-oxo-8-(3-(trifluoromethyl)phenyl)-2,3,5,8-tetrahydro-[1,2,4]triazolo[4,3-a]pyrimidine-5-yl)phenyl)-N,N,N-trimethylethanaminium methanesulfonate dehydrate", "date_of_designation_or_refusal": "22/02/2018", "intended_use": "Treatment of cystic fibrosis", "eu_designation_number": "EU/3/18/1988", "status": "Positive", "first_published_date": "11/04/2018", "last_updated_date": "11/04/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-1988" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "pyridoxal 5'-phosphate", "date_of_designation_or_refusal": "22/02/2018", "intended_use": "Treatment of pyridoxamine 5'-phosphate oxidase deficiency", "eu_designation_number": "EU/3/18/1983", "status": "Positive", "first_published_date": "11/04/2018", "last_updated_date": "11/04/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-18-1983" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "melatonin", "date_of_designation_or_refusal": "13/03/2018", "intended_use": "Treatment of partial deep dermal and full-thickness burns", "eu_designation_number": "-", "status": "Negative", "first_published_date": "11/04/2018", "last_updated_date": "11/04/2018", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/refusal-orphan-designation-treatment-partial-deep-dermal-full-thickness-burns" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "1-[[[4-(4-Fluoro-2-methyl-1H-indol-5-yloxy)-6-methoxyquinolin-7-yl]oxy]methyl]cyclopropanamine-dihydrochloride", "date_of_designation_or_refusal": "22/02/2018", "intended_use": "Treatment of soft tissue sarcoma", "eu_designation_number": "EU/3/18/1972", "status": "Positive", "first_published_date": "11/04/2018", 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"medicine_name": "", "related_ema_product_number": "", "active_substance": "Paromomycin sulfate", "date_of_designation_or_refusal": "11/04/2005", "intended_use": "Treatment of visceral leishmaniasis", "eu_designation_number": "EU/3/05/271", "status": "Withdrawn", "first_published_date": "01/07/2005", "last_updated_date": "18/05/2011", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-05-271" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "recombinant human residue 41 glutamic acid to glutamine variant of interferon alfa-2b", "date_of_designation_or_refusal": "19/01/2009", "intended_use": "Treatment of Behçet's Disease", "eu_designation_number": "EU/3/08/606", "status": "Withdrawn", "first_published_date": "29/06/2009", "last_updated_date": "18/05/2011", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-08-606" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "amonafide L-malate", "date_of_designation_or_refusal": "22/10/2007", "intended_use": "Treatment of acute myeloid leukaemia", "eu_designation_number": "EU/3/07/483", "status": "Withdrawn", "first_published_date": "17/09/2009", "last_updated_date": "18/05/2011", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-07-483" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "26 base single stranded phosphodiester DNA oligonucleotide", "date_of_designation_or_refusal": "08/10/2009", "intended_use": "Treatment of acute myeloid leukaemia", "eu_designation_number": "EU/3/09/662", "status": "Withdrawn", "first_published_date": "20/10/2009", "last_updated_date": "18/05/2011", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-09-662" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "pixantrone 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"eu_designation_number": "EU/3/05/267", "status": "Withdrawn", "first_published_date": "15/10/2009", "last_updated_date": "15/10/2009", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-05-267" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "(Z)-N-[2-(Diethylamino)ethyl]-5-[(5-fluoro-2-oxo-1,2-dihydro-3H-indol-3-ylidene)methyl]-2,4-dimethyl-1H-pyrrole-3-carboxamide (S)-2-hydroxysyccinate", "date_of_designation_or_refusal": "10/03/2005", "intended_use": "Treatment of renal-cell carcinoma", "eu_designation_number": "EU/3/05/268", "status": "Withdrawn", "first_published_date": "15/10/2009", "last_updated_date": "15/10/2009", "orphan_designation_url": "https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-05-268" }, { "medicine_name": "", "related_ema_product_number": "", "active_substance": "4,5-Dihydro-2-(2,4-dihydroxyphenyl)-4-methylthiazole-4(S)-carboxylic acid", 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