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1939 results
Orphan designation status
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Orphan designation: cholic acid for: Treatment of inborn errors in primary bile acid synthesis (updated)
Date of designation: 18/12/2002, Positive, Last updated: 25/09/2023 -
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Orphan designation: 7-Beta-hydroxy cholesteryl-3-beta-oleate for: Treatment of glioma (updated)
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 (updated)
Date of designation: 19/10/2020, Positive, Last updated: 22/09/2023 -
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Orphan designation: Talquetamab for: Treatment of multiple myeloma (updated)
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 (updated)
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 (updated)
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 (updated)
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 (updated)
Date of designation: 19/10/2020, Positive, Last updated: 15/09/2023 -
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Orphan designation: Dabrafenib mesylate for: Treatment of glioma (updated)
Date of designation: 09/12/2020, Positive, Last updated: 15/09/2023 -
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Orphan designation: Asunercept for: Treatment of myelodysplastic syndromes (updated)
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 (updated)
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 (updated)
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 (updated)
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 (updated)
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 (updated)
Date of designation: 05/08/2011, Positive, Last updated: 13/09/2023 -
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Orphan designation: Lentiviral vector carrying the Fanconi anaemia-A (FANCA) gene for: Treatment of Fanconi anaemia type A (updated)
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 (updated)
Date of designation: 15/05/2009, Positive, Last updated: 11/09/2023 -
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Orphan designation: 4-Amino-(6R,S)-5,6,7,8-tetrahydro-L-biopterin dihydrochloride (ronopterin) for: Treatment of moderate and severe traumatic brain injury
Date of designation: 28/08/2006, Positive, Last updated: 11/08/2023 -
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Orphan designation: Recombinant human factor IX protein modified with three point mutations (dalcinonacog alfa) for: Treatment of haemophilia B
Date of designation: 20/06/2017, Positive, Last updated: 03/08/2023 -
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Orphan designation: Marzeptacog alfa (activated) for: Treatment of haemophilia B
Date of designation: 01/04/2019, Positive, Last updated: 03/08/2023 -
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Orphan designation: Ganaxolone for: Treatment of CDKL5 deficiency disorder
Date of designation: 13/11/2019, Positive, Last updated: 31/07/2023 -
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Orphan designation: Mavorixafor for: Treatment of WHIM syndrome
Date of designation: 25/07/2019, Positive, Last updated: 26/07/2023 -
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Orphan designation: Iodine (124I) evuzamitide for: Diagnosis of ATTR amyloidosis
Date of designation: 09/12/2022, Positive, Last updated: 25/07/2023 -
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Orphan designation: Iodine (124I) evuzamitide for: Diagnosis of AL amyloidosis
Date of designation: 11/11/2022, Positive, Last updated: 25/07/2023 -
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Orphan designation: Dersimelagon for: Treatment of erythropoietic protoporphyria
Date of designation: 16/03/2022, Positive, Last updated: 21/07/2023