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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: [Gly2]-recombinant human glucagon-like peptide for: Treatment of short bowel syndrome
Date of first decision: 12/12/2001, Positive, Last updated: 15/08/2016 -
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Orphan designation: Adeno-associated viral vector expressing lipoprotein lipase for: Treatment of lipoprotein-lipase deficiency
Date of first decision: 09/03/2004, Positive, Last updated: 17/10/2013 -
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Orphan designation: Cyclopropane-1,1-dicarboxylic acid [4-(6,7-dimethoxy-quinolin-4-yloxy)-phenyl]-amide (4-fluoro-phenyl)-amide, (L)-malate salt (cabozantinib) for: Treatment of medullary thyroid carcinoma
Date of first decision: 06/02/2009, Positive, Last updated: 12/05/2014 -
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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 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 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: 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: idebenone for: Treatment of Leber's hereditary optic neuropathy
Date of first decision: 16/02/2007, Positive, Last updated: 11/04/2016 -
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Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Ornithine translocase deficiency (hyperornithinaemia-hyperammonaemia homocitrullinuria (HHH) syndrom
Date of first decision: 10/06/2010, Positive, Last updated: 12/07/2019 -
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Orphan designation: Purified bromelain for: Treatment of partial deep dermal and full-thickness burns
Date of first decision: 30/07/2002, Positive, Last updated: 19/09/2013 -
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Orphan designation: Recombinant human tripeptidyl-peptidase 1 for: Treatment of neuronal ceroid lipofuscinosis type 2
Date of first decision: 12/03/2013, Positive, Last updated: 09/02/2016 -
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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 myelodysplastic syndromes
Date of first decision: 22/08/2014, Positive, Last updated: 28/09/2017 -
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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: cholic acid for: Treatment of inborn errors in primary bile acid synthesis
Date of first decision: 18/12/2002, Positive, Last updated: 12/04/2016 -
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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: Ketoconazole for: Treatment of Cushing's syndrome
Date of first decision: 23/04/2012, Positive, Last updated: 22/09/2020 -
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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: (1R,2R)-octanoic acid[2-(2',3'-dihydro-benzo[1,4] dioxin-6'-yl)-2-hydroxy-1-pyrrolidin-1-ylmethyl-ethyl]-amide-L-tartaric acid salt (eliglustat) for: Treatment of Gaucher disease
Date of first decision: 04/12/2007, Positive, Last updated: 09/02/2018 -
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Orphan designation: defibrotide for: Treatment of hepatic veno-occlusive disease
Date of first decision: 29/07/2004, Positive, Last updated: 17/08/2016 -
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Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of carbamoyl-phosphate synthase-1 deficiency
Date of first decision: 03/02/2010, Positive, Last updated: 15/08/2016 -
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Orphan designation: (S)-ethyl 2-amino-3-(4-(2-amino-6((R)-1-(4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl)-2,2,2-trifluoroethoxy)pyrimidin-4-yl)phenyl)propanoate for: Treatment of carcinoid syndrome
Date of first decision: 30/05/2016, Positive, Last updated: 24/10/2017 -
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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: Lutetium (177Lu)-N-[(4,7,10-Tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl-L-cysteinyl-L-threonine-cyclic(2-7)disulfide (lutetium (177Lu) oxodotreotide) for: Treatment of gastro-entero-pancreatic neuroendocrine tumours
Date of first decision: 31/01/2008, Positive, Last updated: 17/01/2018 -
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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