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560 results
Medicine
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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Date of first decision: 10/08/2015, Withdrawn, -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Date of first decision: 09/02/2012, Withdrawn, -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: 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Date of first decision: 23/02/2011, Withdrawn, -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Date of first decision: 12/02/2015, Withdrawn, Last updated: 15/01/2020 -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: Acetylcysteine for: Treatment of idiopathic pulmonary fibrosis
Date of first decision: 26/01/2005, Withdrawn, -
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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Date of first decision: 09/10/2009, Withdrawn, -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: 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Date of first decision: 27/02/2009, Withdrawn, -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: 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Date of first decision: 19/03/2015, Withdrawn, -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Date of first decision: 09/07/2001, Withdrawn, Last updated: 15/10/2009 -
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Orphan designation: Deoxyribose phosphorothioate (5'-tct-ccc-agc-gtg-cgc-cat-3') for: Treatment of chronic lymphocytic leukaemia
Date of first decision: 21/11/2001, Withdrawn, -
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Orphan designation: Synthetic double-stranded oligomer specific to the SERPINA1 gene and containing a cholesterol-conjugated, acyclic nucleobase analogue for: Treatment of congenital alpha-1 antitrypsin deficiency
Date of first decision: 11/01/2016, Withdrawn, Last updated: 17/05/2017 -
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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: Nilotinib hydrochloride monohydrate for: Treatment of gastrointestinal stromal tumours
Date of first decision: 13/04/2007, Withdrawn, -
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Orphan designation: Ethanol (96 per cent) (gel for injection) for: Treatment of congenital venous malformations
Date of first decision: 09/03/2004, Withdrawn, Last updated: 18/01/2016 -
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Orphan designation: Brivanib alaninate for: Treatment of hepatocellular carcinoma
Date of first decision: 27/10/2011, Withdrawn, -
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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 first decision: 10/03/2005, Withdrawn, Last updated: 15/10/2009 -
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Orphan designation: Tralokinumab for: Treatment of idiopathic pulmonary fibrosis
Date of first decision: 08/11/2012, Withdrawn, -
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Orphan designation: Talarozole for: Treatment of autosomal recessive congenital ichthyosis
Date of first decision: 04/07/2012, Withdrawn, Last updated: 19/01/2016 -
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Orphan designation: Monoclonal antibody to human interleukin-6 for: Treatment of post-transplantation lymphoproliferative disorders
Date of first decision: 18/12/2002, Withdrawn, -
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Orphan designation: 4-[2-(6-Methylpyridin-2-yl)-5,6-dihydro-4H-pyrrolo[1,2-b]pyrazol-3-yl]-quinoline-6-carboxamide monohydrate for: Treatment of glioma
Date of first decision: 26/04/2013, Withdrawn, Last updated: 17/06/2019 -
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Orphan designation: Autologous urothelial and smooth muscle cells for: Treatment of spina bifida
Date of first decision: 18/03/2008, Withdrawn, Last updated: 24/05/2012 -
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Orphan designation: Amrubicin hydrochloride for: Treatment of small cell lung cancer
Date of first decision: 02/04/2008, Withdrawn, Last updated: 11/08/2016 -
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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: unoprostone isopropyl for: Treatment of retinitis pigmentosa
Date of first decision: 19/06/2013, Withdrawn, -
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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 renal-cell carcinoma
Date of first decision: 10/03/2005, Withdrawn, Last updated: 15/10/2009