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222 results
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Summary of opinion: Elzonris
tagraxofusp, opinion date: 23/07/2020, Positive, Last updated: 13/11/2020 -
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Summary of opinion: Bluevac BTV (previously known as Bluevac BTV8)
opinion date: 18/06/2020, Positive, Last updated: 19/06/2020 -
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Summary of opinion: Tecartus
opinion date: 15/10/2020, Positive, Last updated: 16/10/2020 -
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Summary of opinion: Blincyto
blinatumomab, opinion date: 15/10/2020, Positive, Last updated: 16/10/2020 -
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Summary of opinion: Symkevi
tezacaftor, ivacaftor, opinion date: 17/09/2020, Positive, Last updated: 18/09/2020 -
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Summary of opinion: Kyprolis
carfilzomib, opinion date: 12/11/2020, Positive, Last updated: 13/11/2020 -
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Summary of opinion: Lumoxiti
moxetumomab pasudotox, opinion date: 10/12/2020, Positive, Last updated: 11/12/2020 -
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Summary of opinion: Inrebic
fedratinib, opinion date: 10/12/2020, Positive, Last updated: 11/12/2020 -
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Summary of opinion: Kalydeco
ivacaftor, opinion date: 17/09/2020, Positive, Last updated: 18/09/2020 -
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Orphan designation: Fenfluramine hydrochloride for: Treatment of Dravet syndrome (updated)
Date of first decision: 18/12/2013, Positive, Last updated: 08/01/2021 -
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Orphan designation: Autologous CD34+ cells transfected with lentiviral vector containing the human arylsulfatase A cDNA for: treatment of metachromatic leukodystrophy (updated)
Date of first decision: 13/04/2007, Positive, Last updated: 22/12/2020 -
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Orphan designation: Treosulfan for: Conditioning treatment prior to haematopoietic-progenitor-cell transplantation
Date of first decision: 22/02/2004, Positive, Last updated: 11/12/2020 -
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Orphan designation: Adeno-associated viral vector serotype 9 containing the human SMN gene (onasemnogene abeparvovec) for: Treatment of spinal muscular atrophy
Date of first decision: 19/06/2015, Positive, Last updated: 10/12/2020 -
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Orphan designation: (S)-2-nitro-6-(4-trifluoromethoxy)benzyloxy)-6,7-dihydro-5H-imidazo[2,1-b] [1,3] oxazine (pretomanid) for: Treatment of tuberculosis
Date of first decision: 29/11/2007, Positive, Last updated: 09/12/2020 -
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Orphan designation: Obiltoxaximab for: Treatment of anthrax
Date of first decision: 24/08/2018, Positive, Last updated: 27/11/2020 -
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues (lumasiran) for: Treatment of primary hyperoxaluria
Date of first decision: 21/03/2016, Positive, Last updated: 25/11/2020 -
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Orphan designation: Humanised monoclonal antibody against P-selectin (crizanlizumab) for: Treatment of sickle cell disease
Date of first decision: 09/08/2012, Positive, Last updated: 24/11/2020 -
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Orphan designation: Recombinant human beta-glucuronidase (vestronidase alfa) for: Treatment of mucopolysaccharidosis type VII (Sly syndrome)
Date of first decision: 21/03/2012, Positive, Last updated: 11/11/2020 -
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Orphan designation: (3S)-3-{4-[7-(aminocarbonyl)-2H-indazol-2-yl] phenyl} piperidine tosylate monohydrate salt (niraparib) for: Treatment of ovarian cancer
Date of first decision: 04/08/2010, Positive, Last updated: 06/11/2020 -
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Orphan designation: Amikacin sulfate for: Treatment of nontuberculous mycobacterial lung disease
Date of first decision: 08/04/2014, Positive, Last updated: 03/11/2020 -
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Orphan designation: N-(2,4-di-tert-butyl-5-hydroxyphenyl)-1,4-dihydro-4-oxoquinoline-3-carboxamide (ivacaftor) for: Treatment of cystic fibrosis
Date of first decision: 08/07/2008, Positive, Last updated: 26/10/2020 -
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Orphan designation: Mepolizumab for: Treatment of hypereosinophilic syndrome
Date of first decision: 29/07/2004, Withdrawn, Last updated: 23/10/2020 -
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Orphan designation: (2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amide (avacopan) for: Treatment of granulomatosis with polyangiitis
Date of first decision: 19/11/2014, Positive, Last updated: 15/10/2020 -
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Orphan designation: (2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amNide for: Treatment of microscopic polyangiitis (avacopan)
Date of first decision: 19/11/2014, Positive, Last updated: 15/10/2020 -
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Orphan designation: Ketoconazole for: Treatment of Cushing's syndrome
Date of first decision: 23/04/2012, Positive, Last updated: 22/09/2020