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2626 results
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Orphan designation: Autologous CD34+ cells transfected with a lentiviral vector containing codon optimised RPS19 gene for: Treatment of Diamond-Blackfan anaemia (updated)
Date of designation: 20/08/2021, Positive, Last updated: 08/06/2022 -
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Orphan designation: polatuzumab vedotin for: Treatment of diffuse large B-cell lymphoma (updated)
Date of designation: 16/04/2018, Positive, Last updated: 08/06/2022 -
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Orphan designation: Trifarotene for: Treatment of autosomal recessive congenital ichthyosis (updated)
Date of designation: 24/03/2020, Withdrawn, Last updated: 07/06/2022 -
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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
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
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
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
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
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
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
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
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
Date of designation: 10/03/2005, Positive, Last updated: 24/05/2022 -
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Orphan designation: tisagenlecleucel for: Treatment of follicular lymphoma
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
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
Date of designation: 26/03/2021, Positive, Last updated: 19/05/2022 -
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Orphan designation: avapritinib for: Treatment of mastocytosis
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
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
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
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
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
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
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
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
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
Date of designation: 30/05/2016, Positive, Last updated: 16/05/2022