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93 results
Medicine
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Orphan designation: Isavuconazonium sulfate for: Treatment of mucormycosis
Date of first decision: 04/06/2014, Positive, Last updated: 21/05/2019 -
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Orphan designation: 1-Deoxygalactonojirimycin hydrochloride for: Treatment of Fabry disease
Date of first decision: 22/05/2006, Positive, Last updated: 21/05/2019 -
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Orphan designation: Phosphorothioate oligonucleotide targeted to apolipoprotein C-III (volanesorsen) for: Treatment of familial chylomicronaemia syndrome
Date of first decision: 19/02/2014, Positive, Last updated: 20/05/2019 -
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Orphan designation: Phosphorothioate oligonucleotide targeted to transthyretin (inotersen) for: Treatment of ATTR amyloidosis
Date of first decision: 26/03/2014, Positive, Last updated: 17/05/2019 -
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Orphan designation: Human coagulation factor X for: Treatment of hereditary factor X deficiency
Date of first decision: 14/09/2007, Positive, Last updated: 26/03/2019 -
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Orphan designation: Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (voretigene neparvovec) for: Treatment of retinitis pigmentosa
Date of first decision: 28/07/2015, Positive, Last updated: 04/03/2019 -
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Orphan designation: Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene (voretigene neparvovec) for: Treatment of Leber's congenital amaurosis
Date of first decision: 02/04/2012, Positive, Last updated: 04/03/2019 -
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Orphan designation: Mogamulizumab for: Treatment of cutaneous T-cell lymphoma
Date of first decision: 14/10/2016, Positive, Last updated: 28/01/2019 -
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Orphan designation: Chimeric monoclonal antibody against GD2 for: Treatment of neuroblastoma
Date of first decision: 08/11/2012, Positive, Last updated: 22/01/2019 -
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Orphan designation: 1-(2,2-difluoro-2H-1,3-benzodioxol-5-yl)-N-{1-[(2R)-2,3-dihydroxypropyl]-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl}cyclopropane-1-carboxamide and ivacaftor (tezacaftor and ivacaftor) for: Treatment of cystic fibrosis
Date of first decision: 27/02/2017, Positive, Last updated: 22/11/2018 -
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Orphan designation: Recombinant human N-acetylgalactosamine-6-sulfatase (elosulfase alfa) for: Treatment of mucopolysaccharidosis, type IVA (Morquio A syndrome)
Date of first decision: 24/07/2009, Positive, Last updated: 24/10/2018 -
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Orphan designation: Recombinant human monoclonal IgG1 antibody for fibroblast growth factor 23 (burosumab) for: Treatment of X-linked hypophosphataemia
Date of first decision: 15/10/2014, Positive, Last updated: 22/10/2018 -
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Orphan designation: Autologous CD34+ cells transfected with retroviral vector containing adenosine deaminase gene for: Treatment of severe combined immunodeficiency (SCID) due to adenosine deaminase (ADA) deficiency
Date of first decision: 26/08/2005, Positive, Last updated: 18/10/2018 -
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Orphan designation: Antisense oligonucleotide targeted to the SMN2 gene (nusinersen) for: Treatment of 5q spinal muscular atrophy
Date of first decision: 02/04/2012, Positive, Last updated: 16/10/2018 -
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Orphan designation: Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain (luspatercept) for: Treatment of beta thalassaemia intermedia and major
Date of first decision: 29/07/2014, Positive, Last updated: 12/10/2018 -
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Orphan designation: Daratumumab
Date of first decision: 17/07/2013, Positive, Last updated: 02/10/2018 -
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Orphan designation: Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor (axicabtagene ciloleucel) for: Treatment of primary mediastinal large B-cell lymphoma
Date of first decision: 09/10/2015, Positive, Last updated: 10/09/2018 -
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Orphan designation: Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3 zeta chimeric antigen receptor (axicabtagene ciloleucel) for: Treatment of diffuse large B-cell lymphoma
Date of first decision: 16/12/2014, Positive, Last updated: 10/09/2018 -
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Orphan designation: Nanobody directed towards the human A1 domain of von Willebrand factor (caplacizumab) for: Treatment of thrombotic thrombocytopenic purpura
Date of first decision: 30/04/2009, Positive, Last updated: 10/09/2018 -
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Orphan designation: Recombinant human beta-glucuronidase (vestronidase alfa) for: Treatment of mucopolysaccharidosis type VII (Sly syndrome)
Date of first decision: 21/03/2012, Positive, -
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Orphan designation: (S)-{8-Fluoro-2-2[4-(3-methoxyphenyl)-1-piperazinyl]-3-[2-methoxy-5-(trifluoromethyl)-phenyl]-3, 4-dihydro-4-quinazolinyl} acetic acid (letermovir) for: Prevention of cytomegalovirus disease in patients with impaired cell-mediated immunity
Date of first decision: 15/04/2011, Positive, Last updated: 28/06/2018 -
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Orphan designation: Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 (tisagenlecleucel) for: Treatment of B-lymphoblastic leukaemia/lymphoma
Date of first decision: 29/04/2014, Positive, Last updated: 13/06/2018 -
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Orphan designation: Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 (tisagenlecleucel) for: Treatment of diffuse large B-cell lymphoma
Date of first decision: 14/10/2016, Positive, Last updated: 13/06/2018 -
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA (also known as patisiran) for: Treatment of transthyretin-mediated amyloidosis
Date of first decision: 06/04/2017, Positive, Last updated: 30/05/2018 -
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Orphan designation: Midostaurin for: Treatment of mastocytosis
Date of first decision: 04/08/2010, Positive, Last updated: 29/05/2018