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277 results
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Orphan designation: Masitinib mesilate for: Treatment of pancreatic cancer
Date of designation: 28/10/2009, Positive, -
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Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of hyperargininaemia
Date of designation: 10/06/2010, Positive, Last updated: 18/11/2022 -
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Orphan designation: Treprostinil diethanolamine for: Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension
Date of designation: 26/08/2005, Positive, Last updated: 13/01/2022 -
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Orphan designation: Caffeine citrate for: Prevention of bronchopulmonary dysplasia
Date of designation: 11/04/2014, Positive, -
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Orphan designation: 1,3-Propanedisulfonic acid, disodium salt for: Treatment of systemic secondary amyloidosis
Date of designation: 31/07/2001, Positive, -
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Orphan designation: Fluocinolone acetonide for: Treatment of non-infectious uveitis affecting the posterior segment of the eye
Date of designation: 07/03/2005, Positive, Last updated: 30/05/2007 -
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Orphan designation: Recombinant human alpha-Mannosidase (velmanase alfa) for: Treatment of alpha-mannosidosis
Date of designation: 26/01/2005, Positive, -
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Orphan designation: Mepolizumab for: Treatment of hypereosinophilic syndrome
Date of designation: 29/07/2004, Withdrawn, Last updated: 23/10/2020 -
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Orphan designation: Taliglucerase alfa for: Treatment of Gaucher disease
Date of designation: 23/03/2010, Positive, Last updated: 10/03/2011 -
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Orphan designation: Human heterologous liver cells for: Treatment of argininosuccinic aciduria
Date of designation: 17/12/2010, Positive, Last updated: 16/08/2016 -
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Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of ornithine carbamoyltransferase deficiency
Date of designation: 10/06/2010, Positive, Last updated: 18/11/2022 -
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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: Nanoliposomal irinotecan for: Treatment of pancreatic cancer
Date of designation: 09/12/2011, Positive, Last updated: 17/12/2018 -
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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: Human heterologous liver cells for: Treatment of carbamoyl-phosphate synthase-1 deficiency
Date of designation: 17/12/2010, Positive, Last updated: 16/08/2016 -
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Orphan designation: Sirolimus for: Treatment of chronic non-infectious uveitis
Date of designation: 30/08/2011, Withdrawn, Last updated: 30/08/2022 -
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Orphan designation: Para-aminosalicylic acid for: Treatment of tuberculosis
Date of designation: 17/12/2010, Positive, Last updated: 08/06/2018 -
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Orphan designation: Bardoxolone methyl for: Treatment of Alport syndrome
Date of designation: 25/05/2018, Positive, Last updated: 30/01/2023 -
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Orphan designation: Aplidine for: Treatment of multiple myeloma
Date of designation: 16/11/2004, Positive, Last updated: 11/10/2005 -
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Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of citrullinaemia type 1
Date of designation: 10/06/2010, Positive, Last updated: 18/11/2022 -
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Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of argininosuccinic aciduria
Date of designation: 10/06/2010, Positive, Last updated: 18/11/2022 -
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Orphan designation: Autologous Tumor-Derived gp96 Heat Shock Protein-Peptide Complex for: Treatment of renal-cell carcinoma
Date of designation: 11/04/2005, Positive, Last updated: 04/01/2006 -
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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