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Referral: Zynteglo
betibeglogene autotemcel, associated names: Zynteglo, Article 20 procedures
Status: European Commission final decision, opinion/position date: 22/07/2021, EC decision date: 16/09/2021, Last updated: 11/10/2021 -
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Referral: Angiotensin-II-receptor antagonists (sartans) containing a tetrazole group
valsartan, candesartan, irbesartan, losartan, olmesartan, associated names: Karvezide, Karvea, Irbesartan/Hydrochlorothiazide Teva, Irbesartan Zentiva (previously Irbesartan Winthrop), Irbesartan Teva, Irbesartan Hydrochlorothiazide Zentiva (previously Irbesartan Hydrochlorothiazide Winthrop), Ifirmasta (previously Irbesartan Krka), Ifirmacombi, Aprovel, Neparvis, Exforge, Exforge HCT, Entresto, Dafiro HCT, Dafiro, Copalia HCT, Copalia, Amlodipine / Valsartan Mylan , CoAprovel, Article 31 referrals
Status: European Commission final decision, opinion/position date: 12/11/2020, EC decision date: 19/02/2021, Last updated: 19/03/2021 -
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Referral: Fluorouracil and fluorouracil related substances (capecitabine, tegafur and flucytosine) containing medicinal products
capecitabine, fluorouracil, tegafur, flucytosine, associated names: Xeloda, Teysuno, Capecitabine Accord, Capecitabine Medac, Capecitabine Teva, Ecansya (previously Capecitabine Krka), Article 31 referrals
Status: European Commission final decision, opinion/position date: 30/04/2020, EC decision date: 07/07/2020, Last updated: 23/07/2020 -
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Referral: Lartruvo
olaratumab, associated names: Lartruvo, Article 20 procedures
Status: European Commission final decision, opinion/position date: 31/01/2019, EC decision date: 19/07/2019, Last updated: 22/08/2019 -
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Referral: Direct-acting antivirals indicated for treatment of hepatitis C (interferon-free)
daclatasvir, dasabuvir, sofosbuvir, ledipasvir, simeprevir, ombitasvir, paritaprevir, ritonavir, associated names: Sovaldi, Olysio, Viekirax, Harvoni, Exviera, Daklinza, Article 20 procedures
Status: European Commission final decision, opinion/position date: 15/12/2016, EC decision date: 23/02/2017, Last updated: 09/03/2017 -
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Referral: Renin-angiotensin-system (RAS)-acting agents
captopril, imidapril, zofenopril, candesartan, delapril, telmisartan, aliskiren, moexipril, enalapril, valsartan, fosinopril, irbesartan, perindopril, quinapril, ramipril, eprosartan, olmesartan, trandolapril, losartan, azilsartan, lisinopril, spirapril, benazepril, cilazapril, associated names: Tolucombi, Telmisartan Teva, Telmisartan Teva Pharma, Tolura, Onduarp, Twynsta, Actelsar HCT, Kinzalkomb, MicardisPlus, PritorPlus, Copalia HCT, Dafiro HCT, Exforge HCT, Rasilamlo, Rasilez, Rasilez HCT, Rasitrio, Edarbi, Ipreziv, Aprovel, Ifirmasta (previously Irbesartan Krka), Irbesartan Teva, Irbesartan Zentiva (previously Irbesartan Winthrop), Karvea, Sabervel, CoAprovel, Ifirmacombi, Irbesartan/Hydrochlorothiazide Teva, Irbesartan Hydrochlorothiazide Zentiva (previously Irbesartan Hydrochlorothiazide Winthrop), Karvezide, Kinzalmono (previously Telmisartan Boehringer Ingelheim Pharma KG), Micardis, Pritor, Telmisartan Actavis, Article 31 referrals
Status: European Commission final decision, opinion/position date: 22/05/2014, EC decision date: 04/09/2014, Last updated: 08/10/2014 -
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Orphan designation: N-(2,4-di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide (ivacaftor) for: Treatment of cystic fibrosis (updated)
Date of designation: 08/07/2008, Positive, Last updated: 25/01/2022 -
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Orphan designation: for: Treatment of cystic fibrosis (updated)
Date of designation: 27/02/2017, Positive, Last updated: 25/01/2022 -
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Orphan designation: Ivacaftor, N-(1,3-dimethyl-1H-pyrazole-4-sulfonyl)-6-[3-(3,3,3-trifluoro-2,2-dimethylpropoxy)-1H-pyrazol-1-yl]-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carboxamide, tezacaftor for: Treatment of cystic fibrosis (updated)
Date of designation: 14/12/2018, Positive, Last updated: 25/01/2022 -
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Orphan designation: Carboxypeptidase G2 (glucarpidase) for: Adjunctive treatment in patients at risk of methotrexate toxicity (updated)
Date of designation: 03/02/2003, Positive, Last updated: 20/01/2022 -
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Orphan designation: artesunate for: Treatment of malaria (updated)
Date of designation: 28/02/2020, Positive, Last updated: 13/01/2022 -
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Orphan designation: 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 for: Treatment of paroxysmal nocturnal haemoglobinuria
Date of designation: 22/05/2017, Positive, Last updated: 16/12/2021 -
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Orphan designation: 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) for: Diagnosis of positive folate-receptor status in ovarian cancer
Date of designation: 10/09/2012, Withdrawn, Last updated: 07/12/2021 -
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Orphan designation: 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) for: Treatment of multiple myeloma
Date of designation: 20/04/2017, Positive, Last updated: 03/12/2021 -
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Orphan designation: (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) for: Treatment of progressive familial intrahepatic cholestasis
Date of designation: 16/01/2014, Positive, Last updated: 03/12/2021 -
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Orphan designation: 1-[4-bromo-5-[1-ethyl-7-(methylamino)-2-oxo-1,2-dihydro-1,6-naphthyridin-3-yl]-2-fluorophenyl]-3-phenylurea for: Treatment of gastrointestinal stromal tumours
Date of designation: 08/11/2017, Positive, Last updated: 23/11/2021 -
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Orphan designation: Setmelanotide for: Treatment of leptin receptor deficiency
Date of designation: 19/11/2018, Positive, Last updated: 12/10/2021 -
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Orphan designation: Setmelanotide for: Treatment of pro-opiomelanocortin deficiency
Date of designation: 14/07/2016, Positive, Last updated: 12/10/2021 -
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Orphan designation: velaglucerase alfa for: Treatment of Gaucher disease
Date of designation: 06/06/2010, Positive, Last updated: 24/09/2021 -
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Orphan designation: Recombinant human parathyroid hormone for: Treatment of hypoparathyroidism
Date of designation: 16/01/2014, Positive, Last updated: 24/09/2021 -
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Orphan designation: Recombinant human IgG1 kappa light chain monoclonal antibody targeting plasma kallikrein (lanadelumab) for: Treatment of hereditary angioedema
Date of designation: 09/10/2015, Positive, Last updated: 20/09/2021 -
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Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of post-polycythaemia vera myelofibrosis
Date of designation: 26/11/2010, Positive, Last updated: 17/09/2021 -
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Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of post-essential thrombocythaemia myelofibrosis
Date of designation: 26/11/2010, Positive, Last updated: 17/09/2021 -
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Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of primary myelofibrosis
Date of designation: 01/10/2010, Positive, Last updated: 17/09/2021 -
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Orphan designation: Adenovirus-mediated herpes-simplex-virus thymidine-kinase (HKSV-tk) gene for: Treatment of high-grade glioma with subsequent use of ganciclovir sodium
Date of designation: 06/02/2002, Positive, Last updated: 16/09/2021