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56 results
Medicine
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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: nilotinib for: Treatment of chronic myeloid leukaemia
Date of first decision: 22/05/2006, Expired, Last updated: 20/01/2020 -
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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: Eculizumab for: Treatment of paroxysmal nocturnal haemoglobinuria
Date of first decision: 17/10/2003, Expired, Last updated: 20/11/2019 -
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Orphan designation: Aztreonam lysinate for: Treatment of Gram-negative bacterial lung infection in cystic fibrosis
Date of first decision: 21/06/2004, Expired, Last updated: 16/10/2019 -
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Orphan designation: Muramyl tripeptide phosphatidyl ethanolamine for: Treatment of osteosarcoma
Date of first decision: 21/06/2004, Expired, Last updated: 11/07/2019 -
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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: Histamine dihydrochloride for: Treatment of acute myeloid leukaemia
Date of first decision: 11/04/2005, Expired, Last updated: 20/02/2019 -
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Orphan designation: miglustat for: Treatment of Niemann-Pick's disease, type C
Date of first decision: 16/02/2006, Expired, Last updated: 20/02/2019 -
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Orphan designation: azacitidine for: Treatment of myelodysplastic syndromes
Date of first decision: 06/02/2002, Expired, Last updated: 20/02/2019 -
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Orphan designation: azacitidine for: Treatment of acute myeloid leukaemia
Date of first decision: 29/11/2007, Expired, Last updated: 20/02/2019 -
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Orphan designation: Rufinamide for: Treatment of Lennox-Gastaut syndrome
Date of first decision: 20/10/2004, Expired, Last updated: 20/02/2019 -
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Orphan designation: Recombinant megakaryopoiesis-stimulating protein for: Treatment of idiopathic thrombocytopenic purpura
Date of first decision: 27/05/2005, Expired, Last updated: 20/02/2019 -
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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: Temsirolimus for: Treatment of renal-cell carcinoma
Date of first decision: 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 first decision: 30/05/2001, 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 first decision: 14/11/2002, Expired, Last updated: 03/10/2017 -
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Orphan designation: nelarabine for: Treatment of acute lymphoblastic leukaemia
Date of first decision: 16/06/2005, 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 first decision: 22/05/2006, Expired, Last updated: 03/10/2017 -
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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: Pegylated recombinant human factor IX (nonacog beta pegol) for: Treatment of haemophilia B
Date of first decision: 15/05/2009, Expired, Last updated: 03/08/2017 -
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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: 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: Iduronate-2-sulfatase for: Treatment of mucopolysaccharidosis type II (Hunter's syndrome)
Date of first decision: 11/12/2001, Expired, Last updated: 08/02/2017 -
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Orphan designation: dasatinib for: Treatment of acute lymphoblastic leukaemia
Date of first decision: 23/12/2005, Expired, Last updated: 08/02/2017