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707 results
Orphan designation status
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Orphan designation: Autologous CD34+ cells transduced ex vivo with a lentiviral vector containing a modified gamma-globin gene for: Treatment of sickle cell disease
Date of designation: 13/11/2020, Withdrawn, Last updated: 16/09/2022 -
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Orphan designation: Sirolimus for: Treatment of chronic non-infectious uveitis
Date of designation: 30/08/2011, Withdrawn, Last updated: 30/08/2022 -
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Orphan designation: Human monoclonal antibody against human interleukin 13 for: Treatment of eosinophilic oesophagitis
Date of designation: 13/11/2013, Withdrawn, Last updated: 30/08/2022 -
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Orphan designation: Melphalan flufenamide for: Treatment of plasma cell myeloma
Date of designation: 19/03/2015, Withdrawn, Last updated: 22/08/2022 -
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Orphan designation: autologous dendritic cells pulsed with killed ovarian cancer cells and matured by TLR3 ligand ex vivo for: Treatment of ovarian cancer
Date of designation: 16/04/2018, Withdrawn, Last updated: 17/08/2022 -
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Orphan designation: Recombinant truncated N-terminal fragment of human lens epithelium-derived growth factor for: Treatment of retinitis pigmentosa
Date of designation: 23/08/2017, Withdrawn, Last updated: 12/08/2022 -
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Orphan designation: allogeneic peripheral blood mononuclear cells incubated ex-vivo with 16, 16-dimethyl prostaglandin E2, dexamethasone for: Treatment in haematopoietic stem cell transplantation
Date of designation: 18/11/2016, Withdrawn, Last updated: 10/08/2022 -
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Orphan designation: Recombinant human alpha-glucosidase conjugated with multiple copies of synthetic bismannose-6-phosphate-tetra-mannose glycan (avalglucosidase alfa) for: Treatment of glycogen storage disease type II (Pompe's disease)
Date of designation: 26/03/2014, Withdrawn, Last updated: 02/08/2022 -
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Orphan designation: Moxetumomab pasudotox for: Treatment of B-lymphoblastic leukaemia/lymphoma
Date of designation: 19/07/2022, Withdrawn, Last updated: 25/07/2022 -
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Orphan designation: arimoclomol citrate for: Treatment of inclusion body myositis
Date of designation: 18/07/2022, Withdrawn, Last updated: 19/07/2022 -
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Orphan designation: Arimoclomol for: Treatment of amyotrophic lateral sclerosis
Date of designation: 18/07/2022, Withdrawn, Last updated: 19/07/2022 -
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Orphan designation: Humanised anti-CD37 monoclonal antibody conjugated to maytansinoid DM1 (naratuximab emtansine) for: Treatment of diffuse large B-cell lymphoma
Date of designation: 18/07/2022, Withdrawn, Last updated: 19/07/2022 -
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Orphan designation: Human autologous mesenchymal adult stem cells extracted from adipose tissue for: Treatment of anal fistula
Date of designation: 26/08/2005, Withdrawn, Last updated: 01/07/2022 -
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Orphan designation: Chimeric locked nucleic acid deoxynucleoside phosphorothioate-linked oligonucleotide inhibitor directed against microRNA-155-5p for: Treatment of cutaneous T-cell lymphoma
Date of designation: 22/05/2017, Withdrawn, Last updated: 22/06/2022 -
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Orphan designation: Methyl 4,6-diamino-2-[1-(2-fluorobenzyl)-1H-pyrazolo[3,4-b]pyridine-3-yl]-5-pyrimidinyl(methyl)carbamate (riociguat) for: Treatment of pulmonary arterial hypertension
Date of designation: 20/12/2007, Withdrawn, Last updated: 14/06/2022 -
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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 primary sclerosing cholangitis
Date of designation: 16/01/2014, Withdrawn, Last updated: 14/06/2022 -
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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 primary biliary cirrhosis
Date of designation: 16/01/2014, Withdrawn, Last updated: 14/06/2022 -
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Orphan designation: Trifarotene for: Treatment of autosomal recessive congenital ichthyosis
Date of designation: 24/03/2020, Withdrawn, Last updated: 07/06/2022 -
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Orphan designation: Inebilizumab for: Treatment of neuromyelitis optica spectrum disorders
Date of designation: 20/03/2017, Withdrawn, Last updated: 19/05/2022 -
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Orphan designation: synthetic human hepcidin for: Treatment of sickle cell disease
Date of designation: 18/11/2016, Withdrawn, Last updated: 18/05/2022 -
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Orphan designation: Synthetic hepcidin for: Treatment of beta thalassaemia intermedia and major
Date of designation: 09/10/2015, Withdrawn, Last updated: 18/05/2022 -
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Orphan designation: Obeticholic acid for: Treatment of primary sclerosing cholangitis
Date of designation: 16/03/2014, Withdrawn, Last updated: 10/05/2022 -
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Orphan designation: N-(2-amino-phenyl)-4-[(4-pyridin-3-yl-pyrimidin-2-ylamino)-methyl] benzamide (mocetinostat) for: Treatment of Hodgkin's lymphoma
Date of designation: 14/09/2007, Withdrawn, Last updated: 10/05/2022 -
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Orphan designation: N- (2-Amino-phenyl)-4-[(4-pyridin-3-yl-pyrimidin-2-ylamino)-methyl] benzamide (mocetinostat) for: Treatment of acute myeloid leukaemia
Date of designation: 31/01/2008, Withdrawn, Last updated: 04/05/2022 -
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Orphan designation: Lisocabtagene maraleucel for: Treatment of primary mediastinal large B-cell lymphoma
Date of designation: 19/11/2018, Withdrawn, Last updated: 04/05/2022