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123 results
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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: Onbevzi
bevacizumab, opinion date: 12/11/2020, Positive, Last updated: 13/11/2020 -
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Summary of opinion: Spravato
esketamine, opinion date: 10/12/2020, Positive, Last updated: 11/12/2020 -
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Summary of opinion: Kixelle
insulin aspart, opinion date: 10/12/2020, Positive, Last updated: 11/12/2020 -
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Summary of opinion: Yuflyma
adalimumab, opinion date: 10/12/2020, Positive, Last updated: 11/12/2020 -
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Summary of opinion: Oyavas
bevacizumab, opinion date: 01/02/2021, Positive, Last updated: 01/02/2021 -
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Summary of opinion: Alymsys
bevacizumab, opinion date: 28/01/2021, Positive, Last updated: 01/02/2021 -
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Summary of opinion: Keytruda
pembrolizumab, opinion date: 28/01/2021, Positive, Last updated: 01/02/2021 -
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Summary of opinion: Sirturo
bedaquiline, opinion date: 28/01/2021, Positive, Last updated: 01/02/2021 -
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Summary of opinion: Tysabri
natalizumab, opinion date: 28/01/2021, Positive, Last updated: 01/02/2021 -
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Summary of opinion: Vaxchora
Cholera vaccine (recombinant, live, oral), opinion date: 28/01/2021, Positive, Last updated: 01/02/2021 -
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Orphan designation: Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain (luspatercept) for: Treatment of beta thalassaemia intermedia and major (updated)
Date of first decision: 29/07/2014, Positive, Last updated: 18/02/2021 -
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Orphan designation: Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain (luspatercept) for: Treatment of myelodysplastic syndromes (updated)
Date of first decision: 22/08/2014, Positive, Last updated: 18/02/2021 -
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Orphan designation: Autologous peripheral blood T cells CD4 and CD8 selected and CD3 and CD28 activated transduced with retroviral vector expressing anti CD19 CD28/CD3-zeta chimeric antigen receptor and cultured (KTE-X19) for: Treatment of mantle cell lymphoma
Date of first decision: 13/11/2019, Positive, Last updated: 25/01/2021 -
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Orphan designation: recombinant human interleukin-3 truncated diphtheria toxin fusion protein for: Treatment of blastic plasmacytoid dendritic cell neoplasm
Date of first decision: 11/11/2015, Positive, Last updated: 25/01/2021 -
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Orphan designation: Fenfluramine hydrochloride for: Treatment of Dravet syndrome
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
Date of first decision: 13/04/2007, Positive, Last updated: 22/12/2020 -
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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: 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