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Orphan designation: (S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine for: Treatment of gastrointestinal stromal tumours
Date of first decision: 17/07/2017, Positive, Last updated: 22/05/2019 -
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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: Glyceryl tri-(4-phenylbutyrate) for: Treatment of hyperargininaemia
Date of first decision: 10/06/2010, Positive, Last updated: 12/07/2019 -
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Orphan designation: 1, 1'-[1,4-Phenylenebis (methylene)]-bis-1,4,8,11-tetraazacyclotetradecane for: Treatment to mobilize progenitor cells prior to stem cell transplantation
Date of first decision: 20/10/2004, Positive, Last updated: 18/08/2008 -
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Orphan designation: Gallium (68Ga)-edotreotide for: Diagnosis of gastro-entero-pancreatic neuroendocrine tumours
Date of first decision: 19/03/2015, Positive, -
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Orphan designation: Recombinant fusion protein linking human coagulation factor IX with human albumin for: Treatment of haemophilia B
Date of first decision: 04/02/2010, Positive, Last updated: 28/03/2018 -
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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: 1,3-Propanedisulfonic acid, disodium salt for: Treatment of systemic secondary amyloidosis
Date of first decision: 31/07/2001, Positive, -
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Orphan designation: Recombinant human alpha-Mannosidase (velmanase alfa) for: Treatment of alpha-mannosidosis
Date of first decision: 26/01/2005, Positive, -
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Orphan designation: Carboxypeptidase G2 for: Adjunctive treatment in patients at risk of methotrexate toxicity
Date of first decision: 03/02/2003, Positive, Last updated: 02/04/2020 -
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Orphan designation: Recombinant human beta-glucuronidase (vestronidase alfa) for: Treatment of mucopolysaccharidosis type VII (Sly syndrome)
Date of first decision: 21/03/2012, Positive, Last updated: 11/11/2020 -
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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: Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene for: Treatment of beta thalassaemia intermedia and major
Date of first decision: 24/01/2013, Positive, Last updated: 03/06/2019 -
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Orphan designation: Cannabidiol for: Treatment of Lennox-Gastaut syndrome
Date of first decision: 20/03/2017, Positive, Last updated: 04/10/2019 -
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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: 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: Eculizumab for: Treatment of atypical haemolytic uraemic syndrome (aHUS)
Date of first decision: 24/07/2009, Positive, Last updated: 20/11/2019 -
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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: Ibrutinib for the treatment of treatment of lymphoplasmacytic lymphoma for: Treatment of lymphoplasmacytic lymphoma
Date of first decision: 29/04/2014, Positive, Last updated: 11/01/2016 -
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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: 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: (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: Glyceryl tri-(4-phenylbutyrate) for: Treatment of ornithine carbamoyltransferase deficiency
Date of first decision: 10/06/2010, Positive, Last updated: 12/07/2019 -
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Orphan designation: Ivosidenib for: Treatment of acute myeloid leukaemia
Date of first decision: 12/12/2016, Positive, Last updated: 04/12/2019 -
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Orphan designation: Levamisol hydrochloride for: Treatment of nephrotic syndrome
Date of first decision: 28/10/2005, Positive, Last updated: 08/08/2006