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54 results
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Orphan designation: Recombinant human alpha-Mannosidase (velmanase alfa) for: Treatment of alpha-mannosidosis
Date of designation: 26/01/2005, Positive, -
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Orphan designation: Recombinant human tripeptidyl-peptidase 1 for: Treatment of neuronal ceroid lipofuscinosis type 2
Date of designation: 12/03/2013, Positive, Last updated: 09/02/2016 -
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Orphan designation: idebenone for: Treatment of Leber's hereditary optic neuropathy
Date of designation: 16/02/2007, Positive, Last updated: 11/04/2016 -
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Orphan designation: [Nle4, D-Phe7]-alfa-melanocyte stimulating hormone (afamelanotide) for: Treatment of erythropoietic protoporphyria
Date of designation: 08/05/2008, Positive, Last updated: 11/04/2016 -
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Orphan designation: Recombinant human tissue non-specific alkaline phosphatase - Fc - deca-aspartate fusion protein for: Treatment of hypophosphatasia
Date of designation: 04/12/2008, Positive, Last updated: 20/11/2019 -
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Orphan designation: 3-[5-(2-Fluoro-phenyl)-[1,2,4]oxadiazole-3-yl]-benzoic acid (ataluren) for: Treatment of Duchenne muscular dystrophy
Date of designation: 27/05/2005, Positive, Last updated: 10/03/2015 -
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Orphan designation: Phosphorothioate oligonucleotide targeted to apolipoprotein C-III (volanesorsen) for: Treatment of familial chylomicronaemia syndrome
Date of designation: 19/02/2014, Positive, Last updated: 02/02/2023 -
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Orphan designation: Ex-vivo-expanded autologous human corneal epithelium-containing stem cells for: Corneal lesions, with associated corneal (limbal) stem cell deficiency, due to ocular burns
Date of designation: 07/11/2008, Positive, Last updated: 23/06/2020 -
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Orphan designation: Obiltoxaximab for: Treatment of anthrax
Date of designation: 24/08/2018, Positive, Last updated: 27/11/2020 -
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Orphan designation: (S)-5-amino-3-(4-((5-fluoro-2-methoxybenzamido)methyl)phenyl)-1-(1,1,1-trifluoropropane-2-yl)-1H-pyrazole-4-carboxamide for: Treatment of mantle cell lymphoma
Date of designation: 21/06/2021, Positive, Last updated: 11/05/2022 -
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Orphan designation: defibrotide for: Treatment of hepatic veno-occlusive disease
Date of designation: 29/07/2004, Positive, Last updated: 17/08/2016 -
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Orphan designation: 6alpha-ethyl-chenodeoxycholic acid for: Treatment of primary biliary cirrhosis
Date of designation: 27/07/2010, Positive, Last updated: 21/11/2022 -
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Orphan designation: Metreleptin for: Treatment of familial partial lipodystrophy
Date of designation: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Orphan designation: Metreleptin for: Treatment of Barraquer-Simons syndrome
Date of designation: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Orphan designation: Metreleptin for: Treatment of Berardinelli-Seip syndrome
Date of designation: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Orphan designation: Metreleptin for: Treatment of Lawrence syndrome
Date of designation: 17/07/2012, Positive, Last updated: 02/04/2020 -
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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 Alagille syndrome
Date of designation: 18/12/2013, Positive, Last updated: 14/12/2022 -
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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: Epcoritamab for: Treatment of diffuse large B-cell lymphoma
Date of designation: 24/02/2022, Positive, Last updated: 13/05/2022 -
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Orphan designation: cholic acid for: Treatment of inborn errors in primary bile acid synthesis
Date of designation: 18/12/2002, Positive, Last updated: 11/11/2022 -
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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: recombinant human interleukin-3 truncated diphtheria toxin fusion protein for: Treatment of blastic plasmacytoid dendritic cell neoplasm
Date of designation: 11/11/2015, Positive, Last updated: 25/01/2021 -
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Orphan designation: (R)-2-Methyl-6-nitro-2-{4-[4-(4-trifluoromethoxyphenoxy)piperidin-1-yl]phenoxymethyl}-2,3-dihydroimidazo[2,1-b]oxazole (delamanid) for: Treatment of tuberculosis
Date of designation: 02/02/2008, Positive, Last updated: 18/06/2014 -
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Orphan designation: (S)-2-nitro-6-(4-trifluoromethoxy)benzyloxy)-6,7-dihydro-5H-imidazo[2,1-b] [1,3] oxazine (pretomanid) for: Treatment of tuberculosis
Date of designation: 29/11/2007, Positive, Last updated: 09/12/2020 -
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Orphan designation: Recombinant IgG degrading enzyme of Streptococcus pyogenes (imlifidase) for: Prevention of graft rejection following solid organ transplantation
Date of designation: 12/01/2017, Positive, Last updated: 28/08/2020