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2421 results
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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Date of first decision: 09/02/2012, Withdrawn, -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: 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: Acetylcysteine for: Treatment of idiopathic pulmonary fibrosis
Date of first decision: 26/01/2005, Withdrawn, -
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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: Masitinib mesilate for: Treatment of pancreatic cancer
Date of first decision: 28/10/2009, Positive, -
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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Date of first decision: 23/02/2011, Withdrawn, -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: 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Date of first decision: 12/02/2015, Withdrawn, Last updated: 15/01/2020 -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: 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: 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Date of first decision: 10/08/2015, Withdrawn, -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: 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: Suberolylanilide hydroxamic acid for: Treatment of cutaneous T-cell lymphoma
Date of first decision: 21/06/2004, Withdrawn, Last updated: 12/03/2013 -
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Orphan designation: 2',3',5'-Tri-O-acetyluridine for: Treatment of 5-fluorouracil overdose
Date of first decision: 15/05/2009, Positive, Last updated: 09/06/2009 -
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Orphan designation: 2-[4-Methoxy-3-(2-m-tolyl-ethoxy)-benzoylamino]-indan-2-carboxylic acid for: Treatment of systemic sclerosis
Date of first decision: 12/03/2013, Positive, Last updated: 18/09/2020 -
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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: Propranolol for: Treatment of soft tissue sarcoma
Date of first decision: 12/12/2016, Positive, -
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Orphan designation: Mixture of two adeno-associated viral vectors serotype 8 containing the 5'-half sequence of human MYO7A gene and the 3'-half sequence of human MYO7A gene for: Treatment of Usher syndrome
Date of first decision: 04/07/2014, Positive, -
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Orphan designation: [N-((2S,3R,3aS,3'R,4a'R,6S,6a'R,6b'S,7aR,12a'S,12b'S,Z)-3,6,11',12b'-tetramethyl-2',3a,3',4,4',4a',5,5',6,6',6a',6b',7,7a,7',8',10',12',12a',12b'-icosahydro-1'H,3H-spiro[furo[3,2-b]pyridine-2,9'-naphtho[2,1-a]azulene]-3'-yl)methanesulfonamide hydrochloride] for: Treatment of chondrosarcoma
Date of first decision: 13/05/2011, Withdrawn, -
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Orphan designation: Polyethylene glycol-modified human recombinant truncated cystathionine beta-synthase for: Treatment of homocystinuria
Date of first decision: 30/05/2016, Positive, Last updated: 23/06/2016 -
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Orphan designation: Idelalisib for: Treatment of splenic marginal-zone lymphoma
Date of first decision: 05/08/2013, Withdrawn, -
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Orphan designation: Repertaxin L-lysine salt for: Prevention of delayed graft function in organ transplant
Date of first decision: 19/09/2001, Positive, -
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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: Trientine tetrahydrochloride for: Treatment of Wilson's disease
Date of first decision: 19/03/2015, Withdrawn, -
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Orphan designation: Pyridoxal 5'-phosphate for: Treatment of pyridoxamine 5'-phosphate oxidase deficiency
Date of first decision: 15/10/2014, Positive, -
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Orphan designation: Recombinant human histone H1.3 and recombinant human N-bis-met-histone H1.3 for: Treatment of acute myeloid leukaemia
Date of first decision: 20/12/2007, Positive, Last updated: 13/03/2013 -
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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