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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: 16/07/2021 -
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Orphan designation: Recombinant human anti-interferon gamma monoclonal antibody (emapalumab) for: Treatment of haemophagocytic lymphohistiocytosis
Date of designation: 09/06/2010, Positive, Last updated: 19/06/2019 -
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Orphan designation: Levamisole (hydrochloride) for: Treatment of nephrotic syndrome
Date of designation: 28/10/2005, Positive, Last updated: 08/08/2006 -
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Orphan designation: Adenovirus-mediated herpes-simplex-virus thymidine-kinase (HKSV-tk) gene for: Treatment of high-grade glioma with subsequent use of ganciclovir sodium
Date of designation: 06/02/2002, Positive, Last updated: 16/09/2021 -
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Orphan designation: Decitabine for: Treatment of acute myeloid leukaemia
Date of designation: 08/06/2006, Positive, Last updated: 19/09/2013 -
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Orphan designation: Amikacin sulfate for: Treatment of nontuberculous mycobacterial lung disease
Date of designation: 08/04/2014, Positive, Last updated: 03/11/2020 -
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Orphan designation: Cysteamine hydrochloride for: Treatment of cystinosis
Date of designation: 07/11/2008, Positive, Last updated: 22/02/2017 -
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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: glibenclamide for: Treatment of neonatal diabetes
Date of designation: 15/01/2016, Positive, Last updated: 04/01/2019 -
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Orphan designation: Osilodrostat for: Treatment of Cushing's syndrome
Date of designation: 15/10/2014, Positive, Last updated: 07/07/2020 -
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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: Recombinant human tissue non-specific alkaline phosphatase - Fc - deca-aspartate fusion protein for: Treatment of hypophosphatasia
Date of designation: 04/12/2008, Positive, Last updated: 20/11/2019 -
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Orphan designation: Recombinant human N-acetylgalactosamine-6-sulfatase (elosulfase alfa) for: Treatment of mucopolysaccharidosis, type IVA (Morquio A syndrome)
Date of designation: 24/07/2009, Positive, Last updated: 24/10/2018 -
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Orphan designation: Chimeric anti-interleukin-6 monoclonal antibody (siltuximab) for: Treatment of Castleman's disease
Date of designation: 30/11/2007, Positive, Last updated: 26/08/2021 -
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Orphan designation: Humanised anti-IL-6 receptor monoclonal antibody (satralizumab) for: Treatment of neuromyelitis optica spectrum disorders
Date of designation: 27/06/2016, Positive, Last updated: 30/06/2021 -
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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 designation: 26/08/2005, Positive, Last updated: 01/04/2014 -
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Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of post-polycythaemia vera myelofibrosis
Date of designation: 26/11/2010, Positive, Last updated: 17/09/2021 -
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Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of post-essential thrombocythaemia myelofibrosis
Date of designation: 26/11/2010, Positive, Last updated: 17/09/2021 -
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Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of primary myelofibrosis
Date of designation: 01/10/2010, Positive, Last updated: 17/09/2021 -
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Orphan designation: 1-[4-bromo-5-[1-ethyl-7-(methylamino)-2-oxo-1,2-dihydro-1,6-naphthyridin-3-yl]-2-fluorophenyl]-3-phenylurea for: Treatment of gastrointestinal stromal tumours
Date of designation: 08/11/2017, Positive, Last updated: 23/11/2021 -
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Orphan designation: Chenodeoxycholic acid for: Treatment of inborn errors in primary bile acid synthesis
Date of designation: 16/12/2014, Positive, Last updated: 29/06/2017 -
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Orphan designation: Recombinant fusion protein linking human coagulation factor IX with human albumin for: Treatment of haemophilia B
Date of designation: 04/02/2010, Positive, Last updated: 28/03/2018 -
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Orphan designation: N-[4-[[(2-amino-3,4-dihydro-4-oxo-6-pteridinyl)methyl]amino]benzoyl]-D-gamma-glutamyl-(2S)-2-amino-beta-alanyl-L-alpha-aspartyl-L-cysteine (etarfolatide) for: Diagnosis of positive folate-receptor status in ovarian cancer
Date of designation: 10/09/2012, Withdrawn, Last updated: 07/12/2021 -
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Orphan designation: Recombinant human beta-glucuronidase (vestronidase alfa) for: Treatment of mucopolysaccharidosis type VII (Sly syndrome)
Date of designation: 21/03/2012, Positive, Last updated: 11/11/2020 -
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Orphan designation: Ex-vivo-expanded autologous human corneal epithelium-containing stem cells for: Corneal lesions, with associated corneal (limbal) stem cell deficiency, due to ocular burns
Date of designation: 07/11/2008, Positive, Last updated: 23/06/2020