For help on how to get the results you want, see our search tips.
227 results
Medicine
Summaries of opinion Remove Summaries of opinion filter
Orphan designations Remove Orphan designations filter
Direct healthcare professional communication Remove Direct healthcare professional communication filter
Medicine type
Orphan medicine Remove Orphan medicine filter
-
List item
Summary of opinion: Pemazyre
pemigatinib, opinion date: 28/01/2021, Positive, Last updated: 26/02/2021 -
List item
Summary of opinion: Sirturo
bedaquiline, opinion date: 28/01/2021, Positive, Last updated: 01/02/2021 -
List item
Summary of opinion: Epidyolex
cannabidiol, opinion date: 25/02/2021, Positive, Last updated: 26/02/2021 -
List item
Summary of opinion: Evrysdi
risdiplam, opinion date: 25/02/2021, Positive, Last updated: 26/02/2021 -
List item
Summary of opinion: Kaftrio (new)
ivacaftor, tezacaftor, elexacaftor, opinion date: 25/03/2021, Positive, Last updated: 26/03/2021 -
List item
Summary of opinion: Kalydeco (new)
ivacaftor, opinion date: 25/03/2021, Positive, Last updated: 26/03/2021 -
List item
Direct healthcare professional communication (DHPC): Polivy (polatuzumab vedotin)
Active substance: polatuzumab vedotin, DHPC type: Quality defect, Last updated: 04/05/2020 -
List item
Direct healthcare professional communication (DHPC): Myalepta (metreleptin): inconsistencies in the package leaflet
Active substance: Metreleptin, DHPC type: Quality defect, Last updated: 18/06/2020 -
List item
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 -
List item
Direct healthcare professional communication (DHPC): Zolgensma (onasemnogene abeparvovec): risk for thrombotic microangiopathy
Active substance: onasemnogene abeparvovec, DHPC type: Type II variation, Last updated: 18/03/2021 -
List item
Direct healthcare professional communication (DHPC): Strimvelis® (autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human adenosine deaminase [ADA] cDNA sequence): first case of lymphoid T cell leukaemia after insertional oncogenesis (new)
Active substance: autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human adenosine deaminase (ADA) cDNA sequence from human haematopoietic stem/progenitor (CD34+) cells, DHPC type: Adverse event, Last updated: 22/03/2021 -
List item
Orphan designation: Somapacitan for: Treatment of growth hormone deficiency (updated)
Date of first decision: 24/08/2018, Positive, Last updated: 15/04/2021 -
List item
Orphan designation: Murine anti-CD22 antibody variable region fused to truncated Pseudomonas exotoxin 38 ( Moxetumomab pasudotox) for: Treatment of hairy cell leukaemia
Date of first decision: 05/12/2008, Positive, Last updated: 09/03/2021 -
List item
Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of post-polycythaemia vera myelofibrosis
Date of first decision: 26/11/2010, Positive, Last updated: 03/03/2021 -
List item
Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of post-essential thrombocythaemia myelofibrosis
Date of first decision: 26/11/2010, Positive, Last updated: 03/03/2021 -
List item
Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of primary myelofibrosis
Date of first decision: 01/10/2010, Positive, Last updated: 03/03/2021 -
List item
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
Date of first decision: 29/07/2014, Positive, Last updated: 18/02/2021 -
List item
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
Date of first decision: 22/08/2014, Positive, Last updated: 18/02/2021 -
List item
Orphan designation: pemigatinib for: Treatment of myeloid/lymphoid neoplasms with eosinophilia and rearrangement of PDGFRA, PDGFRB, or FGFR1, or with PCM1-JAK
Date of first decision: 17/10/2019, Positive, Last updated: 01/02/2021 -
List item
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 -
List item
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 -
List item
Orphan designation: Fenfluramine hydrochloride for: Treatment of Dravet syndrome
Date of first decision: 18/12/2013, Positive, Last updated: 08/01/2021 -
List item
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 -
List item
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 -
List item
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