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2394 results
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Orphan designation: (S)-1-(4-fluorophenyl)-1-(2-(4-(6-(1-methyl-1H-pyrazol-4-yl)pyrrolo[2,1-f][1,2,4]triazin-4-yl)piperazin-yl)pyrimidin-5-yl)ethan-1-amine for: Treatment of gastrointestinal stromal tumours
Date of first decision: 17/07/2017, Positive, Last updated: 22/05/2019 -
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Orphan designation: 2'-deoxyguanosylyl-(3',5'-phosphoryl)-2'-deoxythymidylyl-(3',5'-phosphoryl)- 2'-deoxyguanosylyl-(3',5'-phosphoryl)-2'-deoxycytidylyl-(3',5'-phosphoryl)-2'-deoxycytidylyl -(3',5'-phosphoryl)-2'-deoxycytidylyl-(3',5'-phosphoryl)-2'-deoxyguanosylyl... for: Treatment of diffuse large B-cell lymphoma
Date of first decision: 10/08/2015, Withdrawn, -
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Orphan designation: Sodium phenylbutyrate for: Treatment of ornithine-transcarbamylase deficiency
Date of first decision: 09/02/2012, Withdrawn, -
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Orphan designation: Masitinib mesilate for: Treatment of pancreatic cancer
Date of first decision: 28/10/2009, Positive, -
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Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of hyperargininaemia
Date of first decision: 10/06/2010, Positive, Last updated: 12/07/2019 -
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Orphan designation: Plitidepsin for: Treatment of post-essential thrombocythaemia myelofibrosis
Date of first decision: 23/02/2011, Withdrawn, -
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Orphan designation: Heterologous human adult liver-derived progenitor cells for: Treatment of argininosuccinic aciduria
Date of first decision: 17/07/2013, Positive, -
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Orphan designation: Olaratumab for: Treatment of soft tissue sarcoma
Date of first decision: 12/02/2015, Withdrawn, Last updated: 15/01/2020 -
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Orphan designation: Acetylcysteine for: Treatment of idiopathic pulmonary fibrosis
Date of first decision: 26/01/2005, Withdrawn, -
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Orphan designation: Recombinant protein derived from the saliva of the Ornithodoros moubata tick (nomacopan) for: Treatment of Guillain-Barré syndrome
Date of first decision: 27/06/2016, Positive, Last updated: 26/07/2016 -
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Orphan designation: Ursodeoxycholic acid for: Treatment of Niemann-Pick disease
Date of first decision: 22/05/2017, Positive, Last updated: 25/04/2019 -
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Orphan designation: Methylthioninium for: treatment of frontotemporal dementia with parkinsonism-17
Date of first decision: 26/11/2010, Positive, Last updated: 28/03/2019 -
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Orphan designation: Cladribine for: Treatment of indolent non-Hodgkin's lymphoma
Date of first decision: 18/09/2001, Expired, Last updated: 18/06/2014 -
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Orphan designation: humanised fusion protein consisting of extracellular domain of CD24 linked to IgG1 Fc domain for: Prevention of graft-versus-host disease
Date of first decision: 11/11/2015, Positive, Last updated: 09/11/2020 -
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Orphan designation: 5,6,7,8-Tetrahydrobiopterin for: Treatment of hyperphenylalaninaemia
Date of first decision: 02/10/2003, Positive, Last updated: 23/02/2007 -
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Orphan designation: Recombinant human serum amyloid P for: Prevention of scarring post glaucoma filtration surgery
Date of first decision: 09/10/2009, Withdrawn, -
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Orphan designation: 1, 1'-[1,4-Phenylenebis (methylene)]-bis-1,4,8,11-tetraazacyclotetradecane for: Treatment to mobilize progenitor cells prior to stem cell transplantation
Date of first decision: 20/10/2004, Positive, Last updated: 18/08/2008 -
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Orphan designation: Cyclo-Cys-Gly-Gln-Arg-Glu-Thr-Pro-Glu-Gly-Ala-Glu-Ala-Lys-Pro-Trp-Tyr-Cys for: Treatment of high-altitude pulmonary oedema
Date of first decision: 08/02/2013, Positive, -
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Orphan designation: 2-(2-Methyl-5-nitro-1H-imidazol-1-yl)ethylsulfamide for: Treatment of small cell lung cancer
Date of first decision: 22/08/2014, Positive, -
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Orphan designation: hydrocortisone for: Treatment of adrenal insufficiency
Date of first decision: 20/03/2007, Positive, Last updated: 20/10/2020 -
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Orphan designation: Autologous ex-vivo-expanded peripheral polyclonal lymphocytes enriched in activated natural killer cells for: Treatment of multiple myeloma
Date of first decision: 16/10/2017, Positive, -
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Orphan designation: Allogeneic ex-vivo-expanded umbilical-cord blood cells for: Treatment of acute myeloid leukaemia
Date of first decision: 27/02/2009, Withdrawn, -
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Orphan designation: Eflornithine for: Treatment of familial adenomatous polyposis (FAP)
Date of first decision: 20/09/2010, Positive, Last updated: 29/04/2019 -
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Orphan designation: Trientine tetrahydrochloride for: Treatment of Wilson's disease
Date of first decision: 19/03/2015, Withdrawn, -
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Orphan designation: Thalidomide for: Treatment of graft-versus-host disease
Date of first decision: 09/07/2001, Withdrawn, Last updated: 15/10/2009