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233 results
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Summary of opinion: Pemazyre (updated)
pemigatinib, opinion date: 28/01/2021, Positive, Last updated: 26/02/2021 -
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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: 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: Sogroya
somapacitan, opinion date: 28/01/2021, Positive, Last updated: 01/02/2021 -
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Summary of opinion: Nexpovio
selinexor, 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: Evrysdi
risdiplam, opinion date: 25/02/2021, Positive, Last updated: 26/02/2021 -
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Summary of opinion: Cabometyx
cabozantinib, opinion date: 25/02/2021, Positive, Last updated: 26/02/2021 -
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Summary of opinion: Orladeyo
berotralstat, opinion date: 25/02/2021, Positive, Last updated: 26/02/2021 -
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Summary of opinion: Epidyolex
cannabidiol, opinion date: 25/02/2021, Positive, Last updated: 26/02/2021 -
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Direct healthcare professional communication (DHPC): Esbriet (pirfenidone): Important safety update and new recommendations to prevent Drug-Induced Liver Injury (DILI)
Active substance: Pirfenidone, DHPC type: Type II variation, Last updated: 29/10/2020 -
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Direct healthcare professional communication (DHPC): Myalepta (metreleptin): inconsistencies in the package leaflet
Active substance: Metreleptin, DHPC type: Quality defect, Last updated: 18/06/2020 -
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Direct healthcare professional communication (DHPC): Polivy (polatuzumab vedotin)
Active substance: polatuzumab vedotin, DHPC type: Quality defect, Last updated: 04/05/2020 -
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Direct healthcare professional communication (DHPC): Prevymis (letermovir) concentrate for solution for infusion - Essential to administer through sterile 0.2 micron or 0.22 micron polyethersulfone (PES) in-line filter
Active substance: Letermovir, Last updated: 11/09/2020 -
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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: Somapacitan for: Treatment of growth hormone deficiency (updated)
Date of first decision: 24/08/2018, Positive, Last updated: 01/02/2021 -
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Orphan designation: pemigatinib for: Treatment of myeloid/lymphoid neoplasms with eosinophilia and rearrangement of PDGFRA, PDGFRB, or FGFR1, or with PCM1-JAK (updated)
Date of first decision: 17/10/2019, Positive, Last updated: 01/02/2021 -
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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: 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