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4422 results
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Orphan designation: Setmelanotide for: Treatment of Alström syndrome
Date of first decision: 09/01/2020, Positive, Last updated: 09/11/2020 -
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Orphan designation: Setmelanotide for: Treatment of Bardet-Biedl syndrome
Date of first decision: 21/08/2019, Positive, Last updated: 09/11/2020 -
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Orphan designation: humanised fusion protein consisting of extracellular domain of CD24 linked to IgG1 Fc domain for: Prevention of graft-versus-host disease
Date of first decision: 11/11/2015, Positive, Last updated: 09/11/2020 -
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Orphan designation: (3S)-3-{4-[7-(aminocarbonyl)-2H-indazol-2-yl] phenyl} piperidine tosylate monohydrate salt (niraparib) for: Treatment of ovarian cancer
Date of first decision: 04/08/2010, Positive, Last updated: 06/11/2020 -
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Orphan designation: Amikacin sulfate for: Treatment of nontuberculous mycobacterial lung disease
Date of first decision: 08/04/2014, Positive, Last updated: 03/11/2020 -
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Orphan designation: Amikacin sulfate for: Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis
Date of first decision: 25/07/2006, Positive, Last updated: 03/11/2020 -
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Orphan designation: 5,7-dichloro-2-((ethylamino)methyl)-8-hydroxy-3-methylquinazolin-4(3H)-one mesilate for: Treatment of multiple system atrophy
Date of first decision: 16/12/2019, Positive, Last updated: 27/10/2020 -
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Orphan designation: arimoclomol citrate for: Treatment of Niemann-Pick's disease, type C
Date of first decision: 19/11/2014, Positive, Last updated: 27/10/2020 -
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Orphan designation: Arimoclomol for: Treatment of amyotrophic lateral sclerosis
Date of first decision: 26/10/2006, Positive, Last updated: 27/10/2020 -
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Orphan designation: arimoclomol citrate for: Treatment of inclusion body myositis
Date of first decision: 30/05/2016, Positive, Last updated: 27/10/2020 -
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Orphan designation: N-(2,4-di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide (ivacaftor) for: Treatment of cystic fibrosis
Date of first decision: 08/07/2008, Positive, Last updated: 26/10/2020 -
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Orphan designation: Humanised monoclonal antibody against myostatin (domagrozumab) for: Treatment of Duchenne muscular dystrophy
Date of first decision: 08/02/2013, Withdrawn, Last updated: 23/10/2020 -
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Orphan designation: Mepolizumab for: Treatment of Churg-Strauss syndrome
Date of first decision: 12/03/2013, Withdrawn, Last updated: 23/10/2020 -
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Orphan designation: Mepolizumab for: Treatment of hypereosinophilic syndrome
Date of first decision: 29/07/2004, Withdrawn, Last updated: 23/10/2020 -
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Orphan designation: Adeno-associated virus serotype rh74 containing the human micro-dystrophin gene for: Treatment of Duchenne muscular dystrophy
Date of first decision: 28/02/2020, Positive, Last updated: 22/10/2020 -
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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 first decision: 26/10/2018, Positive, Last updated: 21/10/2020 -
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Orphan designation: hydrocortisone for: Treatment of adrenal insufficiency
Date of first decision: 20/03/2007, Positive, Last updated: 20/10/2020 -
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Orphan designation: Magrolimab for: Treatment of myelodysplastic syndromes
Date of first decision: 26/06/2020, Positive, Last updated: 16/10/2020 -
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Orphan designation: Enzastaurin hydrochloride for: Treatment of diffuse large B-cell lymphoma
Date of first decision: 20/03/2007, Positive, Last updated: 16/10/2020 -
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Orphan designation: Enzastaurin hydrochloride for: Treatment of glioma
Date of first decision: 23/12/2005, Positive, Last updated: 16/10/2020 -
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Orphan designation: somatropin for: AIDS wasting
Date of first decision: 08/08/2000, Withdrawn, Last updated: 15/10/2020 -
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Orphan designation: (2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amide (avacopan) for: Treatment of granulomatosis with polyangiitis
Date of first decision: 19/11/2014, Positive, Last updated: 15/10/2020 -
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Orphan designation: (2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amNide for: Treatment of microscopic polyangiitis (avacopan)
Date of first decision: 19/11/2014, Positive, Last updated: 15/10/2020 -
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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
Date of first decision: 01/04/2019, Positive, Last updated: 15/10/2020 -
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Orphan designation: 2-hydroxymethyl-2-methoxymethyl-1-azabicyclo[2,2,2]octan-3-one for: Treatment of ovarian cancer
Date of first decision: 16/12/2014, Positive, Last updated: 13/10/2020