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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 -
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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 -
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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 -
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Referral: Adakveo (new)
crizanlizumab, associated names: Adakveo, Article 20 procedures
Status: Procedure started, Last updated: 27/01/2023 -
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Orphan designation: Allogeneic Epstein-Barr virus specific cytotoxic T lymphocytes for: Treatment of post-transplantation lymphoproliferative disorders (updated)
Date of designation: 21/03/2016, Positive, Last updated: 12/01/2023 -
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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 -
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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 -
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Orphan designation: mitapivat sulfate for: Treatment of pyruvate kinase deficiency
Date of designation: 22/04/2020, Positive, Last updated: 07/12/2022 -
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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 (brexucabtagene autoleucel) for: Treatment of acute lymphoblastic leukaemia
Date of designation: 19/10/2020, Positive, Last updated: 02/12/2022 -
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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 -
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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 -
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Orphan designation: 6alpha-ethyl-chenodeoxycholic acid for: Treatment of primary biliary cirrhosis
Date of designation: 27/07/2010, Positive, Last updated: 21/11/2022 -
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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 -
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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 -
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Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Ornithine translocase deficiency (hyperornithinaemia-hyperammonaemia homocitrullinuria (HHH) syndrom
Date of designation: 10/06/2010, Positive, Last updated: 18/11/2022 -
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Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of hyperargininaemia
Date of designation: 10/06/2010, Positive, Last updated: 18/11/2022 -
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Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of argininosuccinic aciduria
Date of designation: 10/06/2010, Positive, Last updated: 18/11/2022 -
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Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of citrullinaemia type 1
Date of designation: 10/06/2010, Positive, Last updated: 18/11/2022 -
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Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of ornithine carbamoyltransferase deficiency
Date of designation: 10/06/2010, Positive, Last updated: 18/11/2022 -
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Orphan designation: Glyceryl tri-(4-phenylbutyrate) for: Treatment of carbamoyl-phosphate synthase-1 deficiency
Date of designation: 03/02/2010, Positive, Last updated: 18/11/2022 -
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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 -
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Orphan designation: Burosumab for: Treatment of phosphaturic mesenchymal tumour
Date of designation: 16/04/2018, Positive, Last updated: 08/11/2022 -
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Orphan designation: Infigratinib for: Treatment of cholangiocarcinoma
Date of designation: 21/08/2020, Withdrawn, Last updated: 08/11/2022 -
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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 -
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Orphan designation: [Gly2]-recombinant human glucagon-like peptide for: Treatment of short bowel syndrome
Date of designation: 12/12/2001, Positive, Last updated: 12/10/2022