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2782 results
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Orphan designation: Heterologous human adult liver-derived progenitor cells for: Treatment of argininosuccinic aciduria
Date of designation: 17/07/2013, Positive, -
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Orphan designation: Acetylcysteine for: Treatment of idiopathic pulmonary fibrosis
Date of designation: 26/01/2005, Withdrawn, -
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Orphan designation: Masitinib mesilate for: Treatment of pancreatic cancer
Date of designation: 28/10/2009, Positive, -
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Orphan designation: Plitidepsin for: Treatment of post-essential thrombocythaemia myelofibrosis
Date of designation: 23/02/2011, Withdrawn, -
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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Date of designation: 10/08/2015, Withdrawn, -
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Orphan designation: Suberolylanilide hydroxamic acid for: Treatment of cutaneous T-cell lymphoma
Date of designation: 21/06/2004, Withdrawn, Last updated: 12/03/2013 -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 designation: 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 designation: 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 designation: 20/09/2010, Positive, Last updated: 29/04/2019 -
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Orphan designation: Propranolol for: Treatment of soft tissue sarcoma
Date of designation: 12/12/2016, 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 designation: 13/05/2011, Withdrawn, -
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Orphan designation: Idelalisib for: Treatment of splenic marginal-zone lymphoma
Date of designation: 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 designation: 19/09/2001, Positive, -
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Orphan designation: Ursodeoxycholic acid for: Treatment of Niemann-Pick disease
Date of designation: 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 designation: 19/03/2015, Withdrawn, -
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Orphan designation: Pyridoxal 5'-phosphate for: Treatment of pyridoxamine 5'-phosphate oxidase deficiency
Date of designation: 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 designation: 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 designation: 26/11/2010, Positive, Last updated: 28/03/2019 -
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Orphan designation: Thalidomide for: Treatment of graft-versus-host disease
Date of designation: 09/07/2001, Withdrawn, Last updated: 15/10/2009 -
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Orphan designation: Poly-cyclodextrin-bis-cysteine-PEG3400-camptothecin-conjugate for: Treatment of ovarian cancer
Date of designation: 20/03/2017, Positive, Last updated: 05/05/2017 -
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Orphan designation: 2-((2-ethyl-6-(4-(2-(3-hydroxyazetidin-1-yl)-2-oxoethyl)-piperazin-1-yl)-8-methylimidazo[1,2-alpha]pyridin-3-yl)-(methyl)amino)-4-(4-fluorophenyl)-thiazole-5-carbonitrile (ziritaxestat) for: Treatment of idiopathic pulmonary fibrosis
Date of designation: 29/08/2016, Withdrawn, Last updated: 09/04/2021 -
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Orphan designation: Human MHC non-restricted cytotoxic T-cell line for: Treatment of ovarian cancer
Date of designation: 30/11/2009, Positive, -
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Orphan designation: afamelanotide for: Treatment of solar urticaria
Date of designation: 24/07/2009, Positive, Last updated: 11/04/2016 -
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Orphan designation: ciclosporin for: Prevention of rejection for corneal transplant
Date of designation: 03/11/2006, Withdrawn, Last updated: 15/03/2010 -
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Orphan designation: Nitric oxide for: Treatment of cystic fibrosis
Date of designation: 15/10/2014, Positive,