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Medicine
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Orphan designation: Cladribine for: Treatment of indolent non-Hodgkin's lymphoma
Date of first decision: 18/09/2001, Expired, Last updated: 18/06/2014 -
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Orphan designation: 1,5-(Butylimino)-1,5-dideoxy, D-glucitol for: Treatment of Gaucher disease
Date of first decision: 18/10/2000, Expired, Last updated: 27/02/2013 -
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Orphan designation: Bosentan for: Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension
Date of first decision: 15/02/2001, Expired, Last updated: 27/02/2013 -
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Orphan designation: Anagrelide hydrochloride for: Treatment of essential thrombocythaemia
Date of first decision: 30/12/2000, Expired, Last updated: 08/02/2017 -
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Orphan designation: Zinc acetate dihydrate for: Treatment of Wilson's disease
Date of first decision: 31/07/2001, Expired, Last updated: 06/03/2015 -
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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 first decision: 13/03/2002, Expired, Last updated: 30/09/2016 -
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Orphan designation: Alpha-galactosidase A for: Treatment of Fabry disease
Date of first decision: 08/08/2000, Expired, Last updated: 18/11/2011 -
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Orphan designation: nitisinone for: Treatment of tyrosinaemia type 1
Date of first decision: 30/12/2000, Expired, Last updated: 08/05/2015 -
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Orphan designation: Dexrazoxane for: Treatment of anthracycline extravasations
Date of first decision: 19/09/2001, Expired, Last updated: 30/09/2016 -
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Orphan designation: imatinib mesilate for: Treatment of chronic myeloid leukaemia
Date of first decision: 15/02/2001, Expired, Last updated: 10/02/2015 -
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Orphan designation: Recombinant human acid alpha-glucosidase for: Treatment of glycogen storage disease type II (Pompe's disease)
Date of first decision: 15/02/2001, Expired, Last updated: 10/08/2016 -
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Orphan designation: imatinib mesilate for: Treatment of dermatofibrosarcoma protuberans
Date of first decision: 26/08/2005, Expired, Last updated: 09/02/2015 -
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Orphan designation: Hydroxyurea for: Treatment of sickle cell syndrome
Date of first decision: 09/07/2003, Expired, Last updated: 06/08/2017 -
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Orphan designation: Alpha-galactosidase A for: Treatment of Fabry disease
Date of first decision: 08/08/2000, Expired, Last updated: 18/11/2011 -
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Orphan designation: Sorafenib tosylate for: Treatment of hepatocellular carcinoma
Date of first decision: 11/04/2006, Expired, Last updated: 31/01/2018 -
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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 first decision: 05/02/2002, Expired, Last updated: 10/08/2016 -
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Orphan designation: Pegvisomant for: Treatment of acromegaly
Date of first decision: 15/02/2001, Expired, Last updated: 27/02/2013 -
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Orphan designation: Caffeine citrate for: Treatment of primary apnoea of premature newborns
Date of first decision: 18/02/2003, Expired, Last updated: 11/07/2019 -
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Orphan designation: stiripentol for: Treatment of severe myoclonic epilepsy in infancy
Date of first decision: 06/12/2001, Expired, Last updated: 08/02/2017 -
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Orphan designation: trabectedin for: Treatment of ovarian cancer
Date of first decision: 17/10/2003, Expired, Last updated: 27/11/2019 -
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Orphan designation: icatibant acetate for: Treatment of angioedema
Date of first decision: 18/02/2003, Expired, Last updated: 12/08/2020 -
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Orphan designation: laronidase for: Treatment of mucopolysaccharidosis type I
Date of first decision: 15/02/2001, Expired, Last updated: 08/07/2013 -
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Orphan designation: Mitotane for: Treatment of adrenal cortical carcinoma
Date of first decision: 12/06/2002, Expired, Last updated: 19/06/2014 -
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Orphan designation: imatinib mesilate for: Treatment of acute lymphoblastic leukaemia
Date of first decision: 26/08/2005, Expired, Last updated: 10/02/2015 -
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Orphan designation: N-acetylgalactosamine 4-sulfatase for: Treatment of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)
Date of first decision: 15/02/2001, Expired, Last updated: 10/02/2016