269 results
Categories
Human Remove Human filter
-
List item
Summary of opinion: Plerixafor Accord
plerixafor, opinion date: 13/10/2022, Positive, Last updated: 18/10/2022 -
List item
Summary of opinion: Imcivree
setmelanotide, opinion date: 21/07/2022, Positive, Last updated: 22/07/2022 -
List item
Summary of opinion: Dimethyl fumarate Accord
dimethyl fumarate, opinion date: 15/12/2022, Positive, Last updated: 16/12/2022 -
List item
Summary of opinion: Fintepla
fenfluramine, opinion date: 15/12/2022, Positive, Last updated: 16/12/2022 -
List item
Summary of opinion: Hemgenix
etranacogene dezaparvovec, opinion date: 15/12/2022, Positive, Last updated: 16/12/2022 -
List item
Summary of opinion: Imjudo
tremelimumab, opinion date: 15/12/2022, Positive, Last updated: 16/12/2022 -
List item
Summary of opinion: Pombiliti
cipaglucosidase alfa, opinion date: 15/12/2022, Positive, Last updated: 16/12/2022 -
List item
Summary of opinion: Omblastys
iodine (131I) omburtamab, opinion date: 15/12/2022, Negative, Last updated: 16/12/2022 -
List item
Summary of opinion: Trecondi (new)
treosulfan, opinion date: 26/01/2023, Positive, Last updated: 27/01/2023 -
List item
Summary of opinion: Wakix (new)
pitolisant, opinion date: 26/01/2023, Positive, Last updated: 27/01/2023 -
List item
Summary of opinion: Dapagliflozin Viatris (new)
dapagliflozin, opinion date: 26/01/2023, Positive, Last updated: 27/01/2023 -
List item
Summary of opinion: Reblozyl (new)
luspatercept, opinion date: 26/01/2023, Positive, Last updated: 27/01/2023 -
List item
Summary of opinion: Tolvaptan Accord (new)
tolvaptan, opinion date: 26/01/2023, Positive, Last updated: 27/01/2023 -
List item
Summary of opinion: Sitagliptin / Metformin hydrochloride Sun (new)
sitagliptin, metformin hydrochloride, opinion date: 26/01/2023, Positive, Last updated: 27/01/2023 -
List item
Summary of opinion: Sohonos (new)
palovarotene, opinion date: 26/01/2023, Negative, Last updated: 27/01/2023 -
List item
Orphan designation: Phosphorothioate oligonucleotide targeted to apolipoprotein C-III (volanesorsen) for: Treatment of familial chylomicronaemia syndrome (updated)
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 (updated)
Date of designation: 21/03/2016, Positive, Last updated: 30/01/2023 -
List item
Orphan designation: Bardoxolone methyl for: Treatment of Alport syndrome (updated)
Date of designation: 25/05/2018, 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