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2479 results
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Orphan designation: Allogeneic retinal pigment epithelial cells genetically modified with a non-viral vector to express human alpha-L-iduronidase for: Treatment of mucopolysaccharidosis type I (updated)
Date of designation: 15/10/2021, Positive, Last updated: 24/05/2022 -
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Orphan designation: Allogeneic retinal pigment epithelial cells genetically modified with a non-viral vector to express beta-domain deleted human factor VIII for: Treatment of haemophilia A (updated)
Date of designation: 09/12/2020, Positive, Last updated: 24/05/2022 -
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Orphan designation: Tipifarnib for: Treatment of peripheral T-cell lymphoma (updated)
Date of designation: 19/10/2020, Positive, Last updated: 24/05/2022 -
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Orphan designation: 6-((3S,4S)-4-methyl-1-(pyrimidin-2-yl-methyl)pyrrolidin-3-yl)-3-tetrahydropyran-4-yl-7H-imidazo(1,5-a)pyrazin-8-one for: Treatment of sickle cell disease (updated)
Date of designation: 21/08/2020, Positive, Last updated: 24/05/2022 -
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Orphan designation: 3-(3-(3,5-bis(trifluoromethyl)phenyl)-1h-pyrazol-1-yl)propanoic acid for: Treatment of Stargardt's disease (updated)
Date of designation: 24/04/2019, Positive, Last updated: 24/05/2022 -
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Orphan designation: 6-(R)-methyl-5-O-(5-amino-5,6-dideoxy-alpha-L-talofuranosyl)-paromamine sulfate for: Treatment of cystic fibrosis (updated)
Date of designation: 26/10/2018, Positive, Last updated: 24/05/2022 -
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Orphan designation: 6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-α-L-talofuranosyl)-paromamine sulfate for: Treatment of mucopolysaccharidosis type I (updated)
Date of designation: 22/09/2016, Positive, Last updated: 24/05/2022 -
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Orphan designation: N-methyl-4-({4-[({3-methyl(methylsulfonyl)aminopyrazin-2-yl}methyl)amino]-5-(trifluoromethyl)pyrimidin-2-yl}amino)benzamide hydrochloride (defactinib) for: Treatment of ovarian cancer (updated)
Date of designation: 15/01/2015, Positive, Last updated: 24/05/2022 -
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Orphan designation: Tipifarnib for: Treatment of acute myeloid leukaemia (updated)
Date of designation: 10/03/2005, Positive, Last updated: 24/05/2022 -
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Orphan designation: tisagenlecleucel for: Treatment of follicular lymphoma (updated)
Date of designation: 19/07/2021, Positive, Last updated: 19/05/2022 -
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Orphan designation: Inebilizumab for: Treatment of neuromyelitis optica spectrum disorders (updated)
Date of designation: 20/03/2017, Withdrawn, Last updated: 19/05/2022 -
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Orphan designation: Lefitolimod for: Treatment of small cell lung cancer (updated)
Date of designation: 26/03/2021, Positive, Last updated: 19/05/2022 -
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Orphan designation: avapritinib for: Treatment of mastocytosis (updated)
Date of designation: 26/10/2018, Positive, Last updated: 18/05/2022 -
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Orphan designation: N-Hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide for: Treatment of schwannoma (updated)
Date of designation: 06/06/2012, Positive, Last updated: 18/05/2022 -
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Orphan designation: N-Hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide for: Treatment of neurofibromatosis type 2 (updated)
Date of designation: 26/04/2012, Positive, Last updated: 18/05/2022 -
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Orphan designation: N-Hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide for: Treatment of meningioma (updated)
Date of designation: 06/06/2012, Positive, Last updated: 18/05/2022 -
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Orphan designation: Autologous CD34+ cells transduced with a lentiviral vector encoding glucosylceramidase beta for: Treatment of Gaucher disease (updated)
Date of designation: 21/08/2020, Positive, Last updated: 18/05/2022 -
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Orphan designation: Autologous CD34+ cells transduced with a lentiviral RNA vector that results in integrated cDNA encoding for functional cystinosin for: Treatment of cystinosis (updated)
Date of designation: 19/02/2021, Positive, Last updated: 18/05/2022 -
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Orphan designation: synthetic human hepcidin for: Treatment of sickle cell disease (updated)
Date of designation: 18/11/2016, Withdrawn, Last updated: 18/05/2022 -
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Orphan designation: Synthetic hepcidin for: Treatment of beta thalassaemia intermedia and major (updated)
Date of designation: 09/10/2015, Withdrawn, Last updated: 18/05/2022 -
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Orphan designation: 6-{[(1R,2S)-2-aminocyclohexyl]amino}-7-fluoro-4-(1-methyl-1H-pyrazol-4-yl)-1,2-dihydro-3H-pyrrolo[3,4-c]pyridin-3-one monocitrate (also known as mivavotinib) for: Treatment of acute myeloid leukaemia (updated)
Date of designation: 22/02/2018, Positive, Last updated: 16/05/2022 -
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Orphan designation: pentosan polysulfate sodium for: Treatment of interstitial cystitis (updated)
Date of designation: 30/05/2016, Positive, Last updated: 16/05/2022 -
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Orphan designation: 1-[(3S)-3-{4-amino-3-[(3,5-dimethoxyphenyl)ethynyl]-1H-pyrazolo[3,4-d]pyrimidin-1-yl}pyrrolidin-1-yl]-2-propen-1-one (futibatinib) for: Treatment of biliary tract cancer (updated)
Date of designation: 01/04/2019, Positive, Last updated: 10/05/2022 -
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Orphan designation: Obeticholic acid for: Treatment of primary sclerosing cholangitis (updated)
Date of designation: 16/03/2014, Withdrawn, Last updated: 10/05/2022 -
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Orphan designation: N-(2-amino-phenyl)-4-[(4-pyridin-3-yl-pyrimidin-2-ylamino)-methyl] benzamide (mocetinostat) for: Treatment of Hodgkin's lymphoma (updated)
Date of designation: 14/09/2007, Withdrawn, Last updated: 10/05/2022