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Orphan designation: Pralatrexate for: Treatment of peripheral T-cell lymphoma (nodal, other extranodal and leukaemic/disseminated)
Date of designation: 13/04/2007, Positive, Last updated: 07/04/2011 -
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Orphan designation: Sodium benzoate for: Treatment of citrullinaemia type 1
Date of designation: 14/07/2016, Positive, Last updated: 06/09/2016 -
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Orphan designation: Ketoconazole for: Treatment of Cushing's syndrome
Date of designation: 23/04/2012, Positive, Last updated: 22/09/2020 -
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Orphan designation: Sodium benzoate for: Treatment of argininosuccinic aciduria
Date of designation: 18/11/2016, Positive, Last updated: 14/12/2016 -
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Orphan designation: Deferiprone for: Treatment of neurodegeneration with brain iron accumulation
Date of designation: 27/06/2018, Positive, Last updated: 04/02/2020 -
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Orphan designation: RNA, [P-deoxy-P-(dimethylamino)] (2',3'-dideoxy-2',3'-imino-2',3'-seco) (2'a→5') (C-m5U-C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G), P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N dimethylaminophosphonamidate (Eteplirsen) for: Treatment of Duchenne muscular dystrophy
Date of designation: 03/12/2008, Positive, Last updated: 22/01/2019 -
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Orphan designation: (S)-ethyl 2-amino-3-(4-(2-amino-6((R)-1-(4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl)-2,2,2-trifluoroethoxy)pyrimidin-4-yl)phenyl)propanoate for: Treatment of carcinoid syndrome
Date of designation: 30/05/2016, Positive, Last updated: 28/07/2022 -
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Orphan designation: Nanobody directed towards the human A1 domain of von Willebrand factor (caplacizumab) for: Treatment of thrombotic thrombocytopenic purpura
Date of designation: 30/04/2009, Positive, Last updated: 10/09/2018 -
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Orphan designation: Eflornithine for: Treatment of familial adenomatous polyposis (FAP)
Date of designation: 24/01/2013, Positive, Last updated: 29/04/2019 -
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Orphan designation: Human heterologous liver cells for: Treatment of citrullinaemia type 1
Date of designation: 08/10/2009, Positive, Last updated: 16/08/2016 -
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Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of carbamoyl-phosphate synthase-1 deficiency
Date of designation: 03/02/2010, Positive, Last updated: 18/11/2022 -
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Orphan designation: Sodium benzoate for: Treatment of ornithine transcarbamylase deficiency
Date of designation: 14/07/2016, Positive, Last updated: 06/09/2016 -
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Orphan designation: idebenone for: Treatment of Duchenne muscular dystrophy
Date of designation: 20/03/2007, Positive, Last updated: 11/04/2016 -
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Orphan designation: artesunate for: Treatment of malaria
Date of designation: 28/07/2015, Positive, Last updated: 08/06/2020 -
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Orphan designation: arimoclomol citrate for: Treatment of Niemann-Pick's disease, type C
Date of designation: 19/11/2014, Positive, Last updated: 11/04/2023 -
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Orphan designation: Treprostinil sodium for: Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension
Date of designation: 14/04/2004, Positive, Last updated: 13/02/2023 -
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Orphan designation: Masitinib mesilate for: Treatment of amyotrophic lateral sclerosis
Date of designation: 29/08/2016, Positive, Last updated: 03/10/2016 -
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Orphan designation: Inotuzumab ozogamicin for: Treatment of B-cell acute lymphoblastic leukaemia
Date of designation: 07/06/2013, Positive, Last updated: 25/06/2013 -
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Orphan designation: N-(methyl-diazacyclohexyl-methylbenzamide)-azaphenyl-aminothiopyrrole for: Treatment of mastocytosis
Date of designation: 16/11/2004, Positive, Last updated: 27/10/2005 -
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Orphan designation: Alpha-1 antitrypsin for: Treatment of emphysema secondary to congenital alpha-1 antitrypsin deficiency
Date of designation: 16/11/2004, Positive, Last updated: 07/05/2019 -
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Orphan designation: Recombinant fusion protein consisting of human coagulation factor IX attached to the Fc domain of human IgG1 for: Treatment of haemophilia B (congenital factor IX deficiency)
Date of designation: 08/06/2007, Positive, Last updated: 30/05/2016 -
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Orphan designation: Benzoic acid, sodium salt for: Treatment of non-ketotic hyperglycinaemia
Date of designation: 11/09/2002, Positive, Last updated: 08/01/2003 -
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Orphan designation: Sodium benzoate for: Treatment of N-acetylglutamate synthetase (NAGS) deficiency
Date of designation: 18/11/2016, Positive, Last updated: 14/12/2016 -
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Orphan designation: (-)-17(Cyclopropylmethyl)-1,14 beta-dihydroxy-4,5 alpha-epoxy-6beta-[N-methyl-trans-3-(3-furyl) acrylamido] morphinan hydrochloride (nalfurafine) for: Treatment of uraemic pruritus
Date of designation: 11/09/2002, Positive, Last updated: 14/02/2017 -
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Orphan designation: L-asparaginase encapsulated in erythrocytes for: Treatment of acute lymphoblastic leukaemia
Date of designation: 27/10/2006, Positive, Last updated: 29/07/2008