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251 results
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Orphan designation: Octreotide acetate for: Treatment of acromegaly
Date of designation: 05/08/2013, Positive, Last updated: 21/02/2022 -
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Orphan designation: lonapegsomatropin for: Treatment of growth hormone deficiency
Date of designation: 17/10/2019, Positive, Last updated: 17/02/2022 -
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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 (ivacaftor / tezacaftor / elexacaftor) for: Treatment of cystic fibrosis
Date of designation: 14/12/2018, Positive, Last updated: 25/01/2022 -
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Orphan designation: ivacaftor, tezacaftor for: Treatment of cystic fibrosis
Date of designation: 27/02/2017, 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
Date of designation: 03/02/2003, Positive, Last updated: 20/01/2022 -
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Orphan designation: Treprostinil diethanolamine for: Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension
Date of designation: 26/08/2005, Positive, Last updated: 13/01/2022 -
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Orphan designation: artesunate for: Treatment of malaria
Date of designation: 28/02/2020, Positive, Last updated: 13/01/2022 -
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Orphan designation: Sodium phenylbutyrate, Tauroursodeoxycholic acid for: Treatment of amyotrophic lateral sclerosis
Date of designation: 04/06/2020, Positive, Last updated: 05/01/2022 -
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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: 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: 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 -
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Orphan designation: 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) for: Treatment of mantle cell lymphoma
Date of designation: 13/11/2019, Positive, Last updated: 15/09/2021 -
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Orphan designation: Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (axicabtagene ciloleucel) for: Treatment of primary mediastinal large B-cell lymphoma
Date of designation: 09/10/2015, Positive, Last updated: 15/09/2021 -
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Orphan designation: Selumetinib for: Treatment of neurofibromatosis type 1
Date of designation: 31/07/2018, Positive, Last updated: 15/09/2021 -
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Orphan designation: Modified recombinant human C-type natriuretic peptide (Vosoritide) for: Treatment of achondroplasia
Date of designation: 24/01/2013, Positive, Last updated: 09/09/2021 -
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Orphan designation: Humanised Fc engineered monoclonal antibody against CD19 (tafasitamab) for: Treatment of diffuse large B-cell lymphoma
Date of designation: 15/01/2015, Positive, Last updated: 08/09/2021 -
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Orphan designation: Chimeric monoclonal antibody against GD2 for: Treatment of neuroblastoma
Date of designation: 08/11/2012, Positive, Last updated: 26/08/2021 -
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Orphan designation: Chimeric anti-interleukin-6 monoclonal antibody (siltuximab) for: Treatment of Castleman's disease
Date of designation: 30/11/2007, Positive, Last updated: 26/08/2021 -
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Orphan designation: (2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro-1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid for: Treatment of progressive familial intrahepatic cholestasis
Date of designation: 17/07/2012, Positive, Last updated: 28/07/2021 -
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Orphan designation: Recombinant adeno-associated viral vector containing a codon-optimized Padua derivative of human coagulation factor IX cDNA (etranacogene dezaparvovec) for: Treatment of haemophilia B
Date of designation: 21/03/2018, Positive, Last updated: 01/07/2021