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2832 results
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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: 25/09/2023 -
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Orphan designation: Belzupacap sarotalocan for: Treatment of uveal melanoma
Date of designation: 13/04/2022, Last updated: 22/09/2023 -
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Orphan designation: 7-Beta-hydroxy cholesteryl-3-beta-oleate for: Treatment of glioma
Date of designation: 17/12/2010, Positive, Last updated: 22/09/2023 -
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Orphan designation: (S)-1-(5-((2,3-dihydro-[1,4]dioxino[2,3-b]pyridin-7-yl)sulfonyl)-3,4,5,6-tetrahydropyrrolo[3,4-c]pyrrol-2(1H)-yl)-3-hydroxy-2-phenylpropan-1-one for: Treatment of sickle cell disease
Date of designation: 19/10/2020, Positive, Last updated: 22/09/2023 -
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Orphan designation: Talquetamab for: Treatment of multiple myeloma
Date of designation: 20/08/2021, Positive, Last updated: 21/09/2023 -
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Orphan designation: Celecoxib, Ciprofloxacin for: Treatment of amyotrophic lateral sclerosis
Date of designation: 06/01/2021, Positive, Last updated: 19/09/2023 -
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Orphan designation: 3-{[2,3,5,6-tetrafluoro-3'-(trifluoromethoxy)biphenyl-4-yl]carbamoyl}thiophene-2-carboxylic acid for: Treatment of non-infectious uveitis
Date of designation: 19/06/2015, Positive, Last updated: 18/09/2023 -
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Orphan designation: N-(5-tert-Butylisoxazol-3-yl)-N'-{4-[7-(2-(morpholin-4-yl)ethoxy) imidazo[2,1-b][1,3]benzothiazol-2-yl]phenyl}urea di-hydrochloride salt for: Treatment of acute myeloid leukaemia
Date of designation: 23/03/2009, Positive, Last updated: 15/09/2023 -
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Orphan designation: Poly(oxy-1,2-ethanediyl), alpha-hydro-omega-methoxy, ether with N-[[[2-[[6-[[1-[3-[[3-(2,3-dihydroxypropoxy)propyl]amino]-3-oxopropyl]-2,5-dioxo-3-pyrrolidinyl]thio]hexyl]amino]ethyl]amino]carbonyl]-2-methylalanyl-teriparatide (2:1) for: Treatment of hypoparathyroidism
Date of designation: 19/10/2020, Positive, Last updated: 15/09/2023 -
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Orphan designation: everolimus for: Treatment of tuberous sclerosis
Date of designation: 04/08/2010, Expired, Last updated: 15/09/2023 -
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Orphan designation: Dabrafenib mesylate for: Treatment of glioma
Date of designation: 09/12/2020, Positive, Last updated: 15/09/2023 -
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Orphan designation: zilucoplan for: Treatment of myasthenia gravis
Date of designation: 18/07/2022, Positive, Last updated: 15/09/2023 -
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Orphan designation: Asunercept for: Treatment of myelodysplastic syndromes
Date of designation: 23/08/2017, Positive, Last updated: 15/09/2023 -
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Orphan designation: Recombinant fusion protein consisting of the extracellular portion of CD95 fused to the Fc part of a human IgG1 molecule for: Treatment of glioma
Date of designation: 28/01/2010, Positive, Last updated: 15/09/2023 -
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Orphan designation: Recombinant fusion protein consisting of the extracellular portion of CD95 fused to the Fc part of a human IgG1 molecule for: Prevention of graft-versus-host disease
Date of designation: 31/10/2006, Positive, Last updated: 15/09/2023 -
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Orphan designation: 2-[6-(6,7-Dimethoxyquinolin-3-yl)pyridin-3-yl]-N-[3-(1,1,1-trifluoro-2-methylpropan-2-yl)-1,2-oxazol-5-yl]acetamide (zeteletinib) for: Treatment of medullary thyroid carcinoma
Date of designation: 19/02/2021, Positive, Last updated: 13/09/2023 -
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Orphan designation: Entinostat for: Treatment of Hodgkin's lymphoma
Date of designation: 10/06/2010, Positive, Last updated: 13/09/2023 -
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Orphan designation: Mixture of seven synthetic fragments consisting of p21 RAS peptides for: Treatment of pancreatic cancer
Date of designation: 05/08/2011, Positive, Last updated: 13/09/2023 -
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Orphan designation: Pomalidomide for: Treatment of multiple myeloma
Date of designation: 08/10/2009, Expired, Last updated: 13/09/2023 -
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Orphan designation: Enzastaurin hydrochloride for: Treatment of diffuse large B-cell lymphoma
Date of designation: 20/03/2007, Withdrawn, Last updated: 12/09/2023 -
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Orphan designation: Risdiplam for: Treatment of spinal muscular atrophy
Date of designation: 26/02/2019, Withdrawn, Last updated: 12/09/2023 -
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Orphan designation: Adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human iduronate 2-sulfatase gene for: Treatment of mucopolysaccharidosis type II (Hunter's syndrome)
Date of designation: 17/01/2018, Withdrawn, Last updated: 12/09/2023 -
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Orphan designation: Adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human alpha L-iduronidase gene (devafidugene civaparvovec) for: Treatment of mucopolysaccharidosis type I
Date of designation: 17/01/2018, Withdrawn, Last updated: 12/09/2023 -
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Orphan designation: Lentiviral vector carrying the Fanconi anaemia-A (FANCA) gene for: Treatment of Fanconi anaemia type A
Date of designation: 17/12/2010, Positive, Last updated: 11/09/2023 -
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Orphan designation: 2',3',5'-Tri-O-acetyluridine for: Treatment of 5-fluorouracil overdose
Date of designation: 15/05/2009, Positive, Last updated: 11/09/2023