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230 results
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Orphan designation: Masitinib mesilate for: Treatment of pancreatic cancer
Date of first decision: 28/10/2009, Positive, -
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Orphan designation: Caffeine citrate for: Prevention of bronchopulmonary dysplasia
Date of first decision: 11/04/2014, Positive, -
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Orphan designation: Taliglucerase alfa for: Treatment of Gaucher disease
Date of first decision: 23/03/2010, Positive, Last updated: 10/03/2011 -
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Orphan designation: Treprostinil sodium for: Treatment of chronic thromboembolic pulmonary hypertension
Date of first decision: 08/02/2013, Positive, Last updated: 08/04/2020 -
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Orphan designation: Decitabine for: Treatment of acute myeloid leukaemia
Date of first decision: 08/06/2006, Positive, Last updated: 19/09/2013 -
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Orphan designation: Gilteritinib for: Treatment of acute myeloid leukaemia
Date of first decision: 17/01/2018, Positive, Last updated: 08/11/2019 -
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Orphan designation: Human heterologous liver cells (for infusion) for: Treatment of argininosuccinic aciduria
Date of first decision: 17/12/2010, Positive, Last updated: 16/08/2016 -
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Orphan designation: (R)-2-Methyl-6-nitro-2-{4-[4-(4-trifluoromethoxyphenoxy)piperidin-1-yl]phenoxymethyl}-2,3-dihydroimidazo[2,1-b]oxazole (delamanid) for: Treatment of tuberculosis
Date of first decision: 02/02/2008, Positive, Last updated: 18/06/2014 -
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Orphan designation: Chlormethine for: Treatment of cutaneous T-cell lymphoma
Date of first decision: 22/05/2012, Positive, Last updated: 16/03/2017 -
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Orphan designation: Pirfenidone for: Treatment of idiopathic pulmonary fibrosis
Date of first decision: 16/11/2004, Positive, Last updated: 26/03/2015 -
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Orphan designation: Human heterologous liver cells (for infusion) for: Treatment of carbamoyl-phosphate synthase-1 deficiency
Date of first decision: 17/12/2010, Positive, Last updated: 16/08/2016 -
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Orphan designation: Obinutuzumab for: Treatment of chronic lymphocytic leukaemia
Date of first decision: 10/10/2012, Positive, Last updated: 21/02/2018 -
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Orphan designation: Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib) for: Treatment of chronic myeloid leukaemia
Date of first decision: 02/02/2010, Positive, Last updated: 23/03/2015 -
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Orphan designation: (1R,2S) 6-bromo-alpha-[2-(dimethylamino)ethyl]-2-methoxy-alpha-(1-naphthyl)-beta-phenyl-3-quinolineethanol bedaquiline for: Treatment of tuberculosis
Date of first decision: 26/08/2005, Positive, Last updated: 01/04/2014 -
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Orphan designation: Para-aminosalicylic acid for: Treatment of tuberculosis
Date of first decision: 17/12/2010, Positive, Last updated: 08/06/2018 -
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Orphan designation: idebenone
Date of first decision: 08/03/2004, Positive, Last updated: 11/04/2016 -
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Orphan designation: Adeno-associated viral vector expressing lipoprotein lipase for: Treatment of lipoprotein-lipase deficiency
Date of first decision: 09/03/2004, Positive, Last updated: 17/10/2013 -
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Orphan designation: Human coagulation factor X for: Treatment of hereditary factor X deficiency
Date of first decision: 14/09/2007, Positive, Last updated: 26/03/2019 -
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Orphan designation: Cysteamine hydrochloride for: Treatment of cystinosis
Date of first decision: 15/10/2014, Positive, Last updated: 12/11/2014 -
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Orphan designation: 6alpha-ethyl-chenodeoxycholic acid for: Treatment of primary biliary cirrhosis
Date of first decision: 27/07/2010, Positive, Last updated: 19/12/2016 -
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Orphan designation: carglumic acid for: Treatment of propionic acidaemia
Date of first decision: 07/11/2008, Positive, Last updated: 17/09/2013 -
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Orphan designation: Chimeric anti-interleukin-6 monoclonal antibody (siltuximab) for: Treatment of Castleman's disease
Date of first decision: 30/11/2007, Positive, Last updated: 11/07/2014 -
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Orphan designation: (E)-(1S,4S,10S,21R)-7-[(Z)-ethylidene]-4,21-diisopropyl-2-oxa-12,13-dithia-5,8,20,23- tetraazabicyclo[8.7.6]tricos-16-ene-3,6,9,19,22-pentone for: Treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic/disseminated)
Date of first decision: 28/10/2005, Positive, Last updated: 12/10/2018 -
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Orphan designation: Methyl 4,6-diamino-2-[1-(2-fluorobenzyl)-1H-pyrazolo[3,4-b]pyridine-3-yl]-5-pyrimidinyl(methyl)carbamate (riociguat) for: Treatment of pulmonary arterial hypertension
Date of first decision: 20/12/2007, Positive, Last updated: 05/09/2017 -
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Orphan designation: Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (voretigene neparvovec) for: Treatment of retinitis pigmentosa
Date of first decision: 28/07/2015, Positive, Last updated: 04/03/2019