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2757 results
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Orphan designation: pentosan polysulfate sodium for: Treatment of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)
Date of designation: 21/08/2020, Positive, Last updated: 15/12/2022 -
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Orphan designation: pentosan polysulfate sodium for: Treatment of mucopolysaccharidosis type I
Date of designation: 19/11/2014, Positive, Last updated: 15/12/2022 -
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Orphan designation: edaravone for: Treatment of amyotrophic lateral sclerosis
Date of designation: 16/12/2014, Positive, Last updated: 15/12/2022 -
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Orphan designation: Concizumab for: Treatment of haemophilia B
Date of designation: 12/10/2017, Withdrawn, Last updated: 14/12/2022 -
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Orphan designation: Humanised monoclonal IgG4 antibody against tissue-factor-pathway inhibitor (concizumab) for: Treatment of haemophilia A
Date of designation: 10/10/2012, Withdrawn, Last updated: 14/12/2022 -
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Orphan designation: Alisporivir for: Treatment of collagen VI-related myopathies
Date of designation: 16/03/2022, Positive, Last updated: 14/12/2022 -
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Orphan designation: besilesomab for: Treatment in haematopoietic stem cell transplantation
Date of designation: 17/10/2019, Positive, Last updated: 14/12/2022 -
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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: Parsaclisib for: Treatment of marginal zone lymphoma
Date of designation: 25/07/2019, Withdrawn, Last updated: 13/12/2022 -
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Orphan designation: Tremelimumab for: Treatment of hepatocellular carcinoma
Date of designation: 09/12/2020, Withdrawn, Last updated: 09/12/2022 -
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Orphan designation: 3-[4-(1H-imidazol-1-ylmethyl)phenyl]-5-(2-methylpropyl)thiophene-2-[(N-butyloxylcarbamate)-sulphonamide] sodium salt for: Treatment of idiopathic pulmonary fibrosis
Date of designation: 14/07/2016, Positive, Last updated: 08/12/2022 -
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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: Allogeneic retinal pigment epithelial cells genetically modified with a non-viral vector to express beta-domain deleted human factor VIII for: Treatment of haemophilia A
Date of designation: 09/12/2020, Withdrawn, Last updated: 05/12/2022 -
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Orphan designation: Lentiviral vector encoding human coagulation factor IX for: Treatment of haemophilia B
Date of designation: 26/02/2019, Positive, Last updated: 02/12/2022 -
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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: 1-(3-{4-[3,4-difluoro-2-(trifluoromethyl)phenyl]piperidine-1-carbonyl}-1H,4H,5H,6H,7H-pyrazolo[3,4- c]pyridin-6-yl)ethan-1-one (Tinlarebant) for: Treatment of Stargardt's disease
Date of designation: 25/05/2018, Positive, Last updated: 01/12/2022 -
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Orphan designation: Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene (betibeglogene autotemcel) for: Treatment of beta thalassaemia intermedia and major
Date of designation: 24/01/2013, Withdrawn, Last updated: 30/11/2022 -
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Orphan designation: A combination of H-Lys-Lys-Gly-Pro-Arg-Cys(SH)-Leu-Thr-Arg-Tyr-Tyr-Ser-Ser-Phe-Val-Asn-Met-Glu-Gly-Lys-Lys-OH and H-Lys-Lys-Gly-Asp-Asn-Ile-Met-Val-Thr-Phe-Arg-Asn-Gln-Ala-Ser-Arg-Pro-Tyr-Gly-Lys-Lys-OH for: Treatment of haemophilia A
Date of designation: 19/11/2014, Positive, Last updated: 29/11/2022 -
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Orphan designation: Linerixibat for: Treatment of primary biliary cholangitis
Date of designation: 12/11/2021, Positive, Last updated: 29/11/2022 -
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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: 1-(2-hydroxyethyl)-8-{[5-(4-methylpiperazin-1-yl)-2-(trifluoromethoxy) phenyl]amino}-4,5-dihydro-1H-pyrazolo[4,3-h]quinazoline-3-carboxamide fumarate salt (onvansertib) for: Treatment of acute myeloid leukaemia
Date of designation: 24/08/2018, Withdrawn, Last updated: 22/11/2022 -
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Orphan designation: 5-{(1R,2R)-2-[(cyclopropylmethyl)amino]cyclopropyl}-N-(tetrahydro-2H-pyran-4-yl)thiophene-3-carboxamide monohydrochloride for: Treatment of Kabuki syndrome
Date of designation: 19/11/2018, Withdrawn, Last updated: 22/11/2022 -
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Orphan designation: Hexasodium phytate for: Treatment of calciphylaxis
Date of designation: 17/07/2012, Positive, Last updated: 22/11/2022 -
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Orphan designation: Recombinant chimeric monoclonal antibody against CD20 (ublituximab) for: Treatment of chronic lymphocytic leukaemia
Date of designation: 26/11/2009, Positive, Last updated: 22/11/2022