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56 results
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Orphan designation status
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Orphan designation: Ibuprofen for: Treatment of patent ductus arteriosus
Date of first decision: 15/02/2001, Expired, Last updated: 06/03/2015 -
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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: 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: imatinib mesilate for: Treatment of malignant gastrointestinal stromal tumours
Date of first decision: 20/11/2001, Expired, Last updated: 09/02/2015 -
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Orphan designation: Temsirolimus for: Treatment of mantle cell lymphoma
Date of first decision: 07/11/2006, Expired, Last updated: 18/11/2011 -
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Orphan designation: N-carbamyl-L-glutamic acid for: Treatment of N-acetylglutamate synthetase (NAGS) deficiency
Date of first decision: 18/10/2000, Expired, Last updated: 25/07/2014 -
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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: 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: dasatinib for: Treatment of acute lymphoblastic leukaemia
Date of first decision: 23/12/2005, Expired, Last updated: 08/02/2017 -
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Orphan designation: imatinib mesilate for: Treatment of chronic eosinophilic leukaemia and the hypereosinophilic syndrome
Date of first decision: 28/10/2005, Expired, Last updated: 09/02/2015 -
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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: Arsenic trioxide for: Treatment of acute promyelocytic leukaemia
Date of first decision: 18/10/2000, Expired, Last updated: 17/10/2013 -
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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: iloprost for: Treatment of primary and of the following forms of secondary pulmonary hypertension…
Date of first decision: 30/12/2000, Expired, Last updated: 08/10/2013 -
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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: busulfan for: Conditioning treatment prior to haematopoietic-progenitor-cell transplantation
Date of first decision: 30/12/2000, Expired, Last updated: 31/07/2013 -
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Orphan designation: sildenafil citrate for: Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension
Date of first decision: 12/12/2003, Expired, Last updated: 19/01/2016 -
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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: 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: dasatinib for: Treatment of chronic myeloid leukaemia
Date of first decision: 23/12/2005, Expired, Last updated: 08/02/2017 -
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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: 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: 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: 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: Recombinant megakaryopoiesis-stimulating protein for: Treatment of idiopathic thrombocytopenic purpura
Date of first decision: 27/05/2005, Expired, Last updated: 20/02/2019