For help on how to get the results you want, see our search tips.
130 results
Medicine
Orphan designations Remove Orphan designations filter
Withdrawn applications Remove Withdrawn applications filter
Categories
Human Remove Human filter
-
List item
Withdrawn application: Gavreto
pralsetinib, date of withdrawal: 03/11/2022, Post-authorisation, Last updated: 02/02/2023 -
List item
Withdrawn application: Imcivree
setmelanotide, date of withdrawal: 22/04/2022, Post-authorisation, Last updated: 28/09/2022 -
List item
Withdrawn application: Zydelig
idelalisib, date of withdrawal: 30/01/2018, Post-authorisation, Last updated: 23/02/2018 -
List item
Withdrawn application: Translarna
ataluren, date of withdrawal: 06/03/2017, Post-authorisation, Last updated: 24/03/2017 -
List item
Withdrawn application: Rienso
ferumoxytol, date of withdrawal: 19/01/2015, Post-authorisation, Last updated: 27/02/2015 -
List item
Withdrawn application: Revlimid
lenalidomide, date of withdrawal: 20/06/2012, Post-authorisation, Last updated: 06/07/2012 -
List item
Withdrawn application: Evoltra
clofarabine, date of withdrawal: 18/03/2008, Post-authorisation, Last updated: 19/03/2008 -
List item
Withdrawn application: Glivec
imatinib, date of withdrawal: 18/10/2006, Post-authorisation, Last updated: 23/10/2006 -
List item
Withdrawn application: Tookad
padeliporfin, date of withdrawal: 14/12/2021, Post-authorisation, Last updated: 28/01/2022 -
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 (updated)
Date of designation: 29/07/2014, Positive, Last updated: 21/03/2023 -
List item
Orphan designation: Fenfluramine hydrochloride for: Treatment of Lennox-Gastaut syndrome
Date of designation: 27/02/2017, Positive, Last updated: 07/02/2023 -
List item
Orphan designation: Phosphorothioate oligonucleotide targeted to apolipoprotein C-III (volanesorsen) for: Treatment of familial chylomicronaemia syndrome
Date of designation: 19/02/2014, Positive, Last updated: 02/02/2023 -
List item
Orphan designation: Allogeneic Epstein-Barr virus specific cytotoxic T lymphocytes (Tabelecleucel) for: Treatment of post-transplantation lymphoproliferative disorders
Date of designation: 21/03/2016, Positive, Last updated: 30/01/2023 -
List item
Orphan designation: Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3 zeta chimeric antigen receptor (axicabtagene ciloleucel) for: Treatment of diffuse large B-cell lymphoma
Date of designation: 16/12/2014, Positive, Last updated: 16/12/2022 -
List item
Orphan designation: (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride (maralixibat chloride) for: Treatment of Alagille syndrome
Date of designation: 18/12/2013, Positive, Last updated: 14/12/2022 -
List item
Orphan designation: mitapivat sulfate for: Treatment of pyruvate kinase deficiency
Date of designation: 22/04/2020, Positive, Last updated: 07/12/2022 -
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 (brexucabtagene autoleucel) for: Treatment of acute lymphoblastic leukaemia
Date of designation: 19/10/2020, Positive, Last updated: 02/12/2022 -
List item
Orphan designation: Maribavir for: Treatment of cytomegalovirus disease in patients with impaired cell-mediated immunity
Date of designation: 07/06/2013, Positive, Last updated: 24/11/2022 -
List item
Orphan designation: Maribavir for: Prevention of cytomegalovirus disease in patients with impaired cell-mediated immunity
Date of designation: 18/12/2007, Positive, Last updated: 24/11/2022 -
List item
Orphan designation: 6alpha-ethyl-chenodeoxycholic acid for: Treatment of primary biliary cirrhosis
Date of designation: 27/07/2010, Positive, Last updated: 21/11/2022 -
List item
Orphan designation: Cyclic pyranopterin monophosphate (fosdenopterin) for: Treatment of molybdenum-cofactor deficiency type A
Date of designation: 20/09/2010, Positive, Last updated: 18/11/2022 -
List item
Orphan designation: Humanised IgG4 monoclonal antibody against total complement component 1, subcomponent s (sutimlimab) for: Treatment of autoimmune haemolytic anaemia
Date of designation: 17/02/2016, Positive, Last updated: 18/11/2022 -
List item
Orphan designation: cholic acid for: Treatment of inborn errors in primary bile acid synthesis
Date of designation: 18/12/2002, Positive, Last updated: 11/11/2022 -
List item
Orphan designation: Burosumab for: Treatment of phosphaturic mesenchymal tumour
Date of designation: 16/04/2018, Positive, Last updated: 08/11/2022 -
List item
Orphan designation: synthetic double-stranded siRNA oligonucleotide targeted against transthyretin mRNA, with six phosphorothioate linkages in the backbone, and nine 2'-fluoro and thirty-five 2'-O-methyl nucleoside residues in the sequence, which is covalently linked via a phosphodiester group to a ligand containing three N-acetylgalactosamine residues (Vutrisiran) for: Treatment of transthyretin-mediated amyloidosis (ATTR amyloidosis)
Date of designation: 25/05/2018, Positive, Last updated: 12/10/2022