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Orphan designation: 1-{3-[3-(4-Chlorophenyl)propoxy]propyl}piperidine, hydrochloride for: Treatment of narcolepsy
Date of designation: 10/07/2007, Positive, Last updated: 20/04/2016 -
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Orphan designation: Mercaptopurine for: Treatment of acute lymphoblastic leukaemia
Date of designation: 22/10/2007, Positive, Last updated: 22/01/2014 -
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Orphan designation: Sodium benzoate for: Treatment of carbamoyl-phosphate synthase-1 deficiency
Date of designation: 14/07/2016, Positive, Last updated: 06/09/2016 -
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Orphan designation: 11-(2-pyrrolidin-1-yl-ethoxy)-14,19-dioxa-5,7,26-triaza-tetracyclo[19.3.1.1(2,6).1(8,12)] heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene for: Treatment of post-essential thrombocythaemia myelofibrosis
Date of designation: 25/08/2010, Positive, Last updated: 29/03/2016 -
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Orphan designation: 11-(2-pyrrolidin-1-yl-ethoxy)-14,19-dioxa-5,7,26-triaza-tetracyclo[19.3.1.1(2,6).1(8,12)] heptacosa-1(25),2(26),3,5,8,10,12(27),16,21,23-decaene for: Treatment of primary myelofibrosis
Date of designation: 25/08/2010, Positive, Last updated: 29/03/2016 -
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Orphan designation: Phosphorothioate oligonucleotide targeted to transthyretin (inotersen) for: Treatment of ATTR amyloidosis
Date of designation: 26/03/2014, Positive, Last updated: 17/05/2019 -
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Orphan designation: Sodium benzoate for: Treatment of hyperargininaemia
Date of designation: 14/07/2016, Positive, Last updated: 06/09/2016 -
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Orphan designation: Pegylated recombinant phenylalanine ammonia lyase (pegvaliase) for: Treatment of hyperphenylalaninaemia
Date of designation: 28/01/2010, Positive, Last updated: 29/05/2019 -
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Orphan designation: Human coagulation factor X for: Treatment of hereditary factor X deficiency
Date of designation: 14/09/2007, Positive, Last updated: 26/03/2019 -
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Orphan designation: Sodium benzoate for: Treatment of lysinuric protein intolerance
Date of designation: 29/08/2016, Positive, Last updated: 03/10/2016 -
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Orphan designation: Gusperimus trihydrochloride for: Treatment of Wegener's granulomatosis
Date of designation: 29/03/2001, Positive, Last updated: 18/11/2011 -
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Orphan designation: Cysteamine hydrochloride for: Treatment of cystinosis
Date of designation: 15/10/2014, Positive, Last updated: 12/11/2014 -
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Orphan designation: Ethyl Eicosapentaenoate for: Treatment of Huntington’s disease
Date of designation: 29/12/2000, Positive, Last updated: 04/01/2006 -
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Orphan designation: idebenone for: Treatment of Friedreich's ataxia
Date of designation: 08/03/2004, Positive, Last updated: 11/04/2016 -
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Orphan designation: synthetic double-stranded siRNA oligonucleotide targeted against transthyretin mRNA, with six phosphorothioate linkages in the backbone, and nine 2'-fluoro and thirty-five 2'-O-methyl nucleoside residues in the sequence, which is covalently linked via a phosphodiester group to a ligand containing three N-acetylgalactosamine residues (Vutrisiran) for: Treatment of transthyretin-mediated amyloidosis (ATTR amyloidosis)
Date of designation: 25/05/2018, Positive, Last updated: 12/10/2022 -
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Orphan designation: Budesonide for: Treatment of eosinophilic oesophagitis
Date of designation: 05/08/2013, Positive, Last updated: 18/01/2018 -
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Orphan designation: NGR-human tumour necrosis factor for: Treatment of malignant mesothelioma
Date of designation: 03/06/2008, Positive, Last updated: 29/07/2008 -
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Orphan designation: mifepristone for: Treatment of hypercortisolism (Cushing's syndrome) of endogenous origin
Date of designation: 27/10/2011, Positive, Last updated: 18/03/2022 -
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Orphan designation: Anti epidermal growth factor receptor antibody h-R3 for: Treatment of glioma
Date of designation: 02/09/2004, Positive, Last updated: 12/10/2018 -
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Orphan designation: Macitentan for: Treatment of pulmonary arterial hypertension
Date of designation: 27/09/2011, Positive, Last updated: 12/10/2018 -
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Orphan designation: Cyclopropane-1,1-dicarboxylic acid [4-(6,7-dimethoxy-quinolin-4-yloxy)-phenyl]-amide (4-fluoro-phenyl)-amide, (L)-malate salt (cabozantinib) for: Treatment of medullary thyroid carcinoma
Date of designation: 06/02/2009, Positive, Last updated: 12/05/2014 -
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Orphan designation: Levoglutamide for: Treatment of sickle cell disease
Date of designation: 04/07/2012, Positive, Last updated: 12/07/2019 -
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Orphan designation: sinapultide, Dipalmitoylphosphatidylcholine, palmitoyloleoyl phosphatidylglycerol, palmitic acid for: Respiratory distress syndrome in premature neonates of less than 32 weeks of gestational age
Date of designation: 29/07/2004, Positive, Last updated: 22/05/2019 -
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Orphan designation: (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride (maralixibat chloride) for: Treatment of progressive familial intrahepatic cholestasis
Date of designation: 16/01/2014, Positive, Last updated: 03/12/2021 -
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Orphan designation: Mexiletine hydrochloride for: Treatment of myotonic disorders
Date of designation: 19/11/2014, Positive, Last updated: 13/06/2018