For help on how to get the results you want, see our search tips.
82 results
-
List item
Summary of opinion: Epysqli
opinion date: 30/03/2023, Positive, Last updated: 31/03/2023 -
List item
Summary of opinion: Lytgobi
futibatinib, opinion date: 26/04/2023, Positive, Last updated: 26/04/2023 -
List item
Summary of opinion: Jaypirca
pirtobrutinib, opinion date: 26/04/2023, Positive, Last updated: 26/04/2023 -
List item
Summary of opinion: Sugammadex Piramal
sugammadex, opinion date: 26/04/2023, Positive, Last updated: 26/04/2023 -
List item
Summary of opinion: Vegzelma
bevacizumab, opinion date: 23/06/2022, Positive, Last updated: 21/06/2022 -
List item
Referral: Adakveo (updated)
crizanlizumab, associated names: Adakveo, Article 20 procedures
Status: Opinion provided by Committee for Medicinal Products for Human Use, opinion/position date: 25/05/2023, Last updated: 26/05/2023 -
List item
Referral: Zynteglo
betibeglogene autotemcel, associated names: Zynteglo, Article 20 procedures
Status: European Commission final decision, opinion/position date: 22/07/2021, EC decision date: 16/09/2021, Last updated: 08/07/2022 -
List item
Referral: Lartruvo
olaratumab, associated names: Lartruvo, Article 20 procedures
Status: European Commission final decision, opinion/position date: 31/01/2019, EC decision date: 19/07/2019, Last updated: 22/08/2019 -
List item
Referral: Factor VIII
associated names: Iblias, Elocta, Kovaltry, Nuwiq, Obizur, NovoEight, Voncento, ReFacto AF, Kogenate Bayer, Helixate NexGen, Advate, Article 31 referrals
Status: European Commission final decision, opinion/position date: 14/09/2017, EC decision date: 10/10/2017, Last updated: 05/12/2017 -
List item
Referral: Direct-acting antivirals indicated for treatment of hepatitis C (interferon-free)
daclatasvir, dasabuvir, sofosbuvir, ledipasvir, simeprevir, ombitasvir, paritaprevir, ritonavir, associated names: Sovaldi, Olysio, Viekirax, Harvoni, Exviera, Daklinza, Article 20 procedures
Status: European Commission final decision, opinion/position date: 15/12/2016, EC decision date: 23/02/2017, Last updated: 09/03/2017 -
List item
Orphan designation: 1-[(3S)-3-{4-amino-3-[(3,5-dimethoxyphenyl)ethynyl]-1H-pyrazolo[3,4-d]pyrimidin-1-yl}pyrrolidin-1-yl]-2-propen-1-one (futibatinib) for: Treatment of biliary tract cancer (updated)
Date of designation: 01/04/2019, Positive, Last updated: 08/05/2023 -
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: (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: 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: 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: 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: Recombinant human IgG1 kappa light chain monoclonal antibody targeting plasma kallikrein (lanadelumab) for: Treatment of hereditary angioedema
Date of designation: 09/10/2015, Positive, Last updated: 04/10/2022 -
List item
Orphan designation: Recombinant human parathyroid hormone for: Treatment of hypoparathyroidism
Date of designation: 16/01/2014, Positive, Last updated: 04/10/2022 -
List item
Orphan designation: Adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene for: Treatment of haemophilia A
Date of designation: 21/03/2016, Positive, Last updated: 28/09/2022 -
List item
Orphan designation: Budesonide for: Treatment of primary IgA nephropathy
Date of designation: 18/11/2016, Positive, Last updated: 14/09/2022 -
List item
Orphan designation: recombinant adeno-associated viral vector serotype 2 carrying the gene for the human aromatic L-amino acid decarboxylase protein, Eladocagene exuparvovec for: Treatment of aromatic L-amino acid decarboxylase deficiency
Date of designation: 18/11/2016, Positive, Last updated: 10/08/2022 -
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 designation: 19/06/2015, Positive, Last updated: 25/07/2022 -
List item
Orphan designation: Mosunetuzumab for: Treatment of follicular lymphoma
Date of designation: 12/11/2021, Positive, Last updated: 23/06/2022 -
List item
Orphan designation: Autologous human T cells genetically modified ex-vivo with a lentiviral vector encoding a chimeric antigen receptor for B-cell maturation antigen for: Treatment of multiple myeloma
Date of designation: 28/02/2020, Positive, Last updated: 13/06/2022