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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Date of designation: 26/01/2005, Withdrawn, -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: Plitidepsin for: Treatment of post-essential thrombocythaemia myelofibrosis
Date of designation: 23/02/2011, Withdrawn, -
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Orphan designation: Olaratumab for: Treatment of soft tissue sarcoma
Date of designation: 12/02/2015, Withdrawn, Last updated: 15/01/2020 -
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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Date of designation: 10/08/2015, Withdrawn, -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Date of designation: 21/06/2004, Withdrawn, Last updated: 12/03/2013 -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: [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Date of designation: 13/05/2011, Withdrawn, -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Date of designation: 05/08/2013, Withdrawn, -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: Trientine tetrahydrochloride for: Treatment of Wilson's disease
Date of designation: 19/03/2015, Withdrawn, -
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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Date of designation: 09/07/2001, Withdrawn, Last updated: 15/10/2009 -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: 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: 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: Cladribine for: Treatment of indolent non-Hodgkin's lymphoma
Date of designation: 18/09/2001, Expired, Last updated: 18/06/2014 -
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Orphan designation: (Z)-N-[2-(Diethylamino)ethyl]-5-[(5-fluoro-2-oxo-1,2-dihydro-3H-indol-3-ylidene)methyl]-2,4-dimethyl-1H-pyrrole-3-carboxamide (S)-2-hydroxysyccinate for: Treatment of malignant gastrointestinal stromal tumours
Date of designation: 10/03/2005, Withdrawn, Last updated: 15/10/2009 -
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Orphan designation: Tralokinumab for: Treatment of idiopathic pulmonary fibrosis
Date of designation: 08/11/2012, Withdrawn, -
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Orphan designation: Recombinant human serum amyloid P for: Prevention of scarring post glaucoma filtration surgery
Date of designation: 09/10/2009, Withdrawn, -
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Orphan designation: Vatreptacog alfa (activated) for: Treatment of haemophilia B
Date of designation: 09/08/2012, Withdrawn, -
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Orphan designation: milciclib maleate for: Treatment of malignant thymoma
Date of designation: 08/11/2012, Withdrawn, Last updated: 22/03/2021 -
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Orphan designation: Ethanol for: Treatment of congenital venous malformations
Date of designation: 09/03/2004, Withdrawn, Last updated: 18/01/2016 -
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Orphan designation: Diphenylcyclopropenone for: Treatment of alopecia totalis
Date of designation: 29/06/2006, Withdrawn, Last updated: 19/09/2013 -
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Orphan designation: Recombinant human C1-inhibitor for: Treatment of angioedema caused by C1 inhibitor deficiency
Date of designation: 11/05/2001, Withdrawn, Last updated: 18/11/2010 -
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Orphan designation: Pegylated recombinant factor VIIa for: Treatment of haemophilia B
Date of designation: 03/06/2008, Withdrawn, -
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Orphan designation: Purified inactivated Japanese encephalitis SA14-4-2 virus vaccine for: Prevention of Japanese encephalitis
Date of designation: 24/01/2006, Withdrawn, -
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Orphan designation: unoprostone isopropyl for: Treatment of retinitis pigmentosa
Date of designation: 19/06/2013, Withdrawn, -
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Orphan designation: Nilotinib hydrochloride monohydrate for: Treatment of gastrointestinal stromal tumours
Date of designation: 13/04/2007, Withdrawn, -
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Orphan designation: Recombinant antibody derivative against human CD19 and CD3 (blinatumomab) for: Treatment of chronic lymphocytic leukaemia
Date of designation: 01/12/2003, Withdrawn, Last updated: 11/12/2020