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Orphan designation: Sorafenib tosilate for: Treatment of hepatocellular carcinoma
Date of designation: 11/04/2006, Expired, Last updated: 31/01/2018 -
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Orphan designation: Temsirolimus for: Treatment of renal-cell carcinoma
Date of designation: 06/04/2006, Expired, Last updated: 22/01/2018 -
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Orphan designation: Ecteinascidin 743 (trabectedin) for: Treatment of soft tissue sarcoma
Date of designation: 30/05/2001, Expired, Last updated: 03/10/2017 -
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Orphan designation: nelarabine for: Treatment of acute lymphoblastic leukaemia
Date of designation: 16/06/2005, Expired, Last updated: 03/10/2017 -
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Orphan designation: 5-aminolevulinic acid hydrochloride for: Intra-operative photodynamic diagnosis of residual glioma
Date of designation: 14/11/2002, Expired, Last updated: 03/10/2017 -
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Orphan designation: Mecasermin for: Treatment of primary insulin-like growth factor-1 deficiency due to molecular or genetic defects
Date of designation: 22/05/2006, Expired, Last updated: 03/10/2017 -
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Orphan designation: Hydroxyurea for: Treatment of sickle cell syndrome
Date of designation: 09/07/2003, Expired, Last updated: 06/08/2017 -
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Orphan designation: Pegylated recombinant human factor IX (nonacog beta pegol) for: Treatment of haemophilia B
Date of designation: 15/05/2009, Expired, Last updated: 03/08/2017 -
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Orphan designation: Anagrelide hydrochloride for: Treatment of essential thrombocythaemia
Date of designation: 30/12/2000, Expired, Last updated: 08/02/2017 -
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Orphan designation: stiripentol for: Treatment of severe myoclonic epilepsy in infancy
Date of designation: 06/12/2001, Expired, Last updated: 08/02/2017 -
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Orphan designation: Iduronate-2-sulfatase for: Treatment of mucopolysaccharidosis type II (Hunter's syndrome)
Date of designation: 11/12/2001, Expired, Last updated: 08/02/2017 -
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Orphan designation: dasatinib for: Treatment of chronic myeloid leukaemia
Date of designation: 23/12/2005, Expired, Last updated: 08/02/2017 -
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Orphan designation: dasatinib for: Treatment of acute lymphoblastic leukaemia
Date of designation: 23/12/2005, Expired, Last updated: 08/02/2017 -
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Orphan designation: 4-(3,5-Bis(hydroxy-phenyl)-1,2,4) triazol-1-yl) benzoic acid for: Treatment of chronic iron overload requiring chelation therapy
Date of designation: 13/03/2002, Expired, Last updated: 30/09/2016 -
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Orphan designation: Dexrazoxane for: Treatment of anthracycline extravasations
Date of designation: 19/09/2001, Expired, Last updated: 30/09/2016 -
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Orphan designation: 2-Chloro-9-[2-deoxy-2-fluoro-ß-D-arabinofuranosyl]adenine for: Treatment of acute lymphoblastic leukaemia
Date of designation: 05/02/2002, Expired, Last updated: 10/08/2016 -
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Orphan designation: Sorafenib tosilate for: Treatment of renal-cell carcinoma
Date of designation: 29/07/2004, Expired, Last updated: 10/08/2016 -
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Orphan designation: Recombinant human acid alpha-glucosidase for: Treatment of glycogen storage disease type II (Pompe's disease)
Date of designation: 15/02/2001, Expired, Last updated: 10/08/2016 -
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Orphan designation: N-acetylgalactosamine 4-sulfatase for: Treatment of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)
Date of designation: 15/02/2001, Expired, Last updated: 10/02/2016 -
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Orphan designation: sildenafil citrate for: Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension
Date of designation: 12/12/2003, Expired, Last updated: 19/01/2016 -
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Orphan designation: nitisinone for: Treatment of tyrosinaemia type 1
Date of designation: 30/12/2000, Expired, Last updated: 08/05/2015 -
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Orphan designation: ziconotide for: Treatment of chronic pain requiring intraspinal analgesia
Date of designation: 09/07/2001, Expired, Last updated: 08/05/2015 -
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Orphan designation: Zinc acetate dihydrate for: Treatment of Wilson's disease
Date of designation: 31/07/2001, Expired, Last updated: 06/03/2015 -
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Orphan designation: Ibuprofen for: Treatment of patent ductus arteriosus
Date of designation: 15/02/2001, Expired, Last updated: 06/03/2015 -
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Orphan designation: imatinib mesilate for: Treatment of chronic myeloid leukaemia
Date of designation: 15/02/2001, Expired, Last updated: 10/02/2015