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1673 results
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Orphan designation: Ciprofloxacin (liposomal) for: Treatment of cystic fibrosis (updated)
Date of first decision: 24/07/2009, Positive, Last updated: 26/06/2020 -
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Orphan designation: Ex-vivo-expanded autologous human corneal epithelium-containing stem cells for: Corneal lesions, with associated corneal (limbal) stem cell deficiency, due to ocular burns (updated)
Date of first decision: 07/11/2008, Positive, Last updated: 23/06/2020 -
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Orphan designation: 2'-O-methyl phosphorothioate RNA oligonucleotide, 5'-m5CUGm5CUGm5CUGm5CUGm5CUGm5CUGm5CUG-3' for: Treatment of Huntington’s disease (updated)
Date of first decision: 12/02/2015, Positive, Last updated: 23/06/2020 -
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Orphan designation: Humanised monoclonal antibody against CD38 for the treatment of plasma cell myeloma (isatuximab) for: Treatment of plasma cell myeloma (updated)
Date of first decision: 29/04/2014, Positive, Last updated: 12/06/2020 -
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Orphan designation: propagermanium for: Treatment of focal segmental glomerulosclerosis (updated)
Date of first decision: 19/11/2018, Positive, Last updated: 11/06/2020 -
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Orphan designation: Modified adenovirus serotype 5/35 containing a CMV promoter-driven transgene cassette with the human transgenes for a membrane-bound CD40 ligand and full length 4-1BBL for: Treatment of pancreatic cancer (updated)
Date of first decision: 28/07/2015, Positive, Last updated: 11/06/2020 -
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Orphan designation: Human monoclonal antibody against Fas ligand for: Treatment of pemphigus (updated)
Date of first decision: 09/02/2012, Positive, Last updated: 11/06/2020 -
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Orphan designation: Sirolimus for: Treatment of sickle cell disease (updated)
Date of first decision: 17/01/2018, Positive, Last updated: 08/06/2020 -
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Orphan designation: Trehalose for: Treatment of spinocerebellar ataxia (updated)
Date of first decision: 19/06/2015, Positive, Last updated: 08/06/2020 -
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Orphan designation: Etamsylate for: Treatment of hereditary haemorrhagic telangiectasia (updated)
Date of first decision: 19/11/2018, Positive, Last updated: 08/06/2020 -
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Orphan designation: 4,6,4'-trimethylangelicin for: Treatment of cystic fibrosis (updated)
Date of first decision: 19/06/2013, Positive, Last updated: 08/06/2020 -
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Orphan designation: Artesunate for: Treatment of malaria (updated)
Date of first decision: 28/07/2015, Positive, Last updated: 08/06/2020 -
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Orphan designation: Sirolimus for: Treatment of beta thalassaemia intermedia and major (updated)
Date of first decision: 14/12/2015, Positive, Last updated: 08/06/2020 -
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Orphan designation: (2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidine-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridine-2-yl)-4-fluoropyrrolidine-2-carboxamide for: Treatment of C3 glomerulopathy (updated)
Date of first decision: 21/03/2018, Positive, Last updated: 08/06/2020 -
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Orphan designation: (2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidin-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridin-2-yl)-4-fluoropyrrolidine-2-carboxamide for: Treatment of paroxysmal nocturnal haemoglobinuria (updated)
Date of first decision: 12/12/2017, Positive, Last updated: 08/06/2020 -
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Orphan designation: Ile-Ser-Ile-Thr-Glu-Ile-Lys-Gly-Val-Ile-Val-His-Arg-Ile-Glu-Thr-Ile-Leu-Phe-Lys-Lys-Lys-Lys-Glu-Met-Pro-Ser-Glu-Glu-Gly-Tyr-Gln-Asp for: Treatment of multiple system atrophy (updated)
Date of first decision: 19/11/2018, Positive, Last updated: 08/06/2020 -
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Orphan designation: 2-((4S)-6-(4-chlorophenyl)-1-methyl-4H-benzo[C]isoxazolo[4,5-e]azepin-4-yl)acetamide monohydrate for: Treatment of myelofibrosis (updated)
Date of first decision: 28/02/2020, Positive, Last updated: 08/06/2020 -
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Orphan designation: Recombinant modified ricin toxin A-chain subunit for: Prevention of ricin poisoning (updated)
Date of first decision: 21/03/2018, Positive, Last updated: 08/06/2020 -
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Orphan designation: Beclomethasone 17, 21-dipropionate (oral use) (beclometasone dipropionate) for: Treatment of intestinal graft-versus-host disease (updated)
Date of first decision: 13/03/2002, Positive, Last updated: 08/06/2020 -
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Orphan designation: Trehalose for: Treatment of oculopharyngeal muscular dystrophy (updated)
Date of first decision: 21/05/2015, Positive, Last updated: 08/06/2020 -
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Orphan designation: vaccine consisting of 5 survivin peptides with different human leukocyte antigen restrictions for: Treatment of ovarian cancer (updated)
Date of first decision: 18/11/2016, Positive, Last updated: 08/06/2020 -
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Orphan designation: Recombinant human pentraxin-2 for: Treatment of primary myelofibrosis (updated)
Date of first decision: 19/11/2014, Positive, Last updated: 08/06/2020 -
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Orphan designation: Recombinant human pentraxin-2 for: Treatment of post-essential thrombocythaemia myelofibrosis (updated)
Date of first decision: 19/11/2014, Positive, Last updated: 08/06/2020 -
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Orphan designation: Recombinant human pentraxin-2 for: Treatment of post-polycythaemia vera myelofibrosis (updated)
Date of first decision: 19/11/2014, Positive, Last updated: 08/06/2020 -
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Orphan designation: Recombinant chimeric monoclonal antibody against CD20 (ublituximab) for: Treatment of chronic lymphocytic leukaemia (updated)
Date of first decision: 26/11/2009, Positive, Last updated: 08/06/2020