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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, Last updated: 11/11/2020 -
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Orphan designation: Recombinant human alpha-Mannosidase (velmanase alfa) for: Treatment of alpha-mannosidosis
Date of first decision: 26/01/2005, Positive, -
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Orphan designation: Metreleptin for: Treatment of familial partial lipodystrophy
Date of first decision: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Orphan designation: Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene for: Treatment of beta thalassaemia intermedia and major
Date of first decision: 24/01/2013, Positive, Last updated: 03/06/2019 -
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Orphan designation: Brentuximab vedotin for: Treatment of cutaneous T-cell lymphoma
Date of first decision: 11/01/2012, Positive, Last updated: 26/08/2020 -
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Orphan designation: (R)-2-Methyl-6-nitro-2-{4-[4-(4-trifluoromethoxyphenoxy)piperidin-1-yl]phenoxymethyl}-2,3-dihydroimidazo[2,1-b]oxazole (delamanid) for: Treatment of tuberculosis
Date of first decision: 02/02/2008, Positive, Last updated: 18/06/2014 -
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Orphan designation: Humanised monoclonal antibody against P-selectin (crizanlizumab) for: Treatment of sickle cell disease
Date of first decision: 09/08/2012, Positive, Last updated: 24/11/2020 -
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Orphan designation: N-methyl D-(2,3,4,5,6-pentahydroxy-hexyl)-ammonium; 2-(3,5-dichloro-phenyl)-benzoxazole-6-carboxylate (tafamidis) for: Treatment of familial amyloid polyneuropathy
Date of first decision: 28/08/2006, Positive, Last updated: 25/02/2020 -
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Orphan designation: "Synthetic 47-amino-acid N-myristoylated lipopeptide, derived from the preS region of hepatitis B virus (bulevirtide) for: Treatment of hepatitis delta virus infection
Date of first decision: 19/06/2015, Positive, Last updated: 12/08/2020 -
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Orphan designation: (1R,2S) 6-bromo-alpha-[2-(dimethylamino)ethyl]-2-methoxy-alpha-(1-naphthyl)-beta-phenyl-3-quinolineethanol bedaquiline for: Treatment of tuberculosis
Date of first decision: 26/08/2005, Positive, Last updated: 01/04/2014 -
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Orphan designation: Metreleptin for: Treatment of Barraquer-Simons syndrome
Date of first decision: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Orphan designation: Recombinant IgG degrading enzyme of Streptococcus pyogenes (imlifidase) for: Prevention of graft rejection following solid organ transplantation
Date of first decision: 12/01/2017, Positive, Last updated: 28/08/2020 -
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Orphan designation: 6alpha-ethyl-chenodeoxycholic acid for: Treatment of primary biliary cirrhosis
Date of first decision: 27/07/2010, Positive, Last updated: 19/12/2016 -
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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: recombinant human interleukin-3 truncated diphtheria toxin fusion protein for: Treatment of blastic plasmacytoid dendritic cell neoplasm
Date of first decision: 11/11/2015, Positive, Last updated: 25/01/2021 -
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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: Humanised monoclonal antibody targeting B-cell maturation antigen conjugated with maleimidocaproyl monomethyl auristatin F for: Treatment of multiple myeloma
Date of first decision: 16/10/2017, Positive, Last updated: 17/09/2020 -
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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: 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: 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: Metreleptin for: Treatment of Berardinelli-Seip syndrome
Date of first decision: 17/07/2012, Positive, Last updated: 02/04/2020 -
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Orphan designation: 2,2'-{2-[(1R)-1-({[(2,5-dichlorobenzoyl)amino]acetyl}amino)-3-methylbutyl]-5-oxo-1,3,2-dioxaborolane-4,4-diyl}diacetic acid for: Treatment of multiple myeloma
Date of first decision: 27/09/2011, Positive, Last updated: 26/08/2020 -
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Orphan designation: polatuzumab vedotin for: Treatment of diffuse large B-cell lymphoma
Date of first decision: 16/04/2018, Positive, Last updated: 27/01/2020 -
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Orphan designation: monoclonal antibody against human CD30 covalently linked to the cytotoxin monomethylauristatin E (brentuximab vedotin) for: Adjunctive treatment in haematopoietic cell transplantation
Date of first decision: 15/01/2009, Positive, Last updated: 26/08/2020 -
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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