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278 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: 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: 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Date of designation: 29/07/2004, Withdrawn, Last updated: 23/10/2020 -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: Ivosidenib for: Treatment of acute myeloid leukaemia
Date of designation: 12/12/2016, Positive, Last updated: 08/06/2021 -
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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, Positive, Last updated: 21/09/2011 -
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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: 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: 27000 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: 27000 for: Treatment of primary myelofibrosis
Date of designation: 25/08/2010, Positive, Last updated: 29/03/2016 -
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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: 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
Date of designation: 08/03/2004, Positive, Last updated: 11/04/2016 -
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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: 09/02/2016 -
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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