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2783 results
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Orphan designation: Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain (luspatercept) for: Treatment of beta thalassaemia intermedia and major (updated)
Date of designation: 29/07/2014, Positive, Last updated: 21/03/2023 -
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Orphan designation: Autologous T-cells transduced with a lentiviral vector encoding a chimeric antigen receptor against CD7 for: Treatment of acute lymphoblastic leukaemia (updated)
Date of designation: 09/12/2022, Positive, Last updated: 14/03/2023 -
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Orphan designation: Panobinostat for: Treatment of multiple myeloma (updated)
Date of designation: 08/11/2012, Positive, Last updated: 13/03/2023 -
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Orphan designation: Lentiviral vector containing the human MYO7A gene for: Treatment of retinitis pigmentosa in Usher syndrome 1B (updated)
Date of designation: 23/03/2010, Positive, Last updated: 13/03/2023 -
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Orphan designation: Lentiviral vector containing the human ABCA4 gene for: Treatment of Stargardt's disease (updated)
Date of designation: 02/02/2010, Positive, Last updated: 13/03/2023 -
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Orphan designation: clofazimine for: Treatment of nontuberculous mycobacterial lung disease (updated)
Date of designation: 21/08/2019, Positive, Last updated: 13/03/2023 -
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Orphan designation: 5-aminolevulinic acid for: Treatment of glioma (updated)
Date of designation: 12/01/2017, Positive, Last updated: 10/03/2023 -
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Orphan designation: Cannabidiol for: Treatment of 22q11.2 deletion syndrome (updated)
Date of designation: 11/11/2022, Positive, Last updated: 07/03/2023 -
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Orphan designation: Cannabidiol for: Treatment of fragile X syndrome (FXS) (updated)
Date of designation: 25/02/2022, Positive, Last updated: 07/03/2023 -
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Orphan designation: 3,5-diamino-6-chloro-N-(N-(4-(4-(2-(hexyl((2S,3R,4R,5R)-2,3,4,5,6-pentahydroxyhexyl)amino)ethoxy)phenyl)butyl)-carbamimidoyl)pyrazine-2-carboxamide, Sodium chloride solution 4.2% (w/v) for: Treatment of primary ciliary dyskinesia (updated)
Date of designation: 16/11/2020, Positive, Last updated: 07/03/2023 -
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Orphan designation: arimoclomol citrate for: Treatment of Niemann-Pick's disease, type C (updated)
Date of designation: 19/11/2014, Positive, Last updated: 01/03/2023 -
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Orphan designation: apomorphine hydrochloride for: Treatment of off-periods in Parkinson's disease not responding to oral treatment (updated)
Date of designation: 16/02/2006, Positive, Last updated: 28/02/2023 -
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Orphan designation: Ribitol for: Treatment of limb-girdle muscular dystrophy (updated)
Date of designation: 19/10/2020, Positive, Last updated: 27/02/2023 -
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against SERPINA1 mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues for: Treatment of congenital alpha-1 antitrypsin deficiency (updated)
Date of designation: 16/12/2019, Positive, Last updated: 24/02/2023 -
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against lactate dehydrogenase A mRNA and containing four modified nucleosides which form a ligand cluster of four N-acetylgalactosamine residues for: Treatment of primary hyperoxaluria (updated)
Date of designation: 31/07/2018, Positive, Last updated: 24/02/2023 -
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Orphan designation: Patidegib for: Treatment of naevoid basal-cell carcinoma syndrome (Gorlin syndrome) (updated)
Date of designation: 21/03/2018, Positive, Last updated: 24/02/2023 -
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Orphan designation: Recombinant human ADAMTS-13 for: Treatment of thrombotic thrombocytopenic purpura (updated)
Date of designation: 03/12/2008, Positive, Last updated: 24/02/2023 -
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Orphan designation: 2-hydroxy-N,N,N-trimethylethan-1-aminium (Z)-4-(5-((3-benzyl-4-oxo-2-thioxothiazolidin-5-ylidene)methyl)furan-2-yl)benzoate for: Treatment of pancreatic cancer
Date of designation: 24/03/2020, Withdrawn, Last updated: 16/02/2023 -
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Orphan designation: Omigapil maleate for: Treatment of congenital muscular dystrophy with merosin (laminin alpha 2) deficiency
Date of designation: 08/05/2008, Positive, Last updated: 15/02/2023 -
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Orphan designation: proteolytic enzymes enriched in bromelain for: Treatment of partial deep dermal and full-thickness burns
Date of designation: 30/07/2002, Expired, Last updated: 15/02/2023 -
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Orphan designation: Treprostinil sodium for: Treatment of pulmonary arterial hypertension and chronic thromboembolic pulmonary hypertension
Date of designation: 14/04/2004, Positive, Last updated: 13/02/2023 -
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Orphan designation: Treprostinil diethanolamine for: Treatment of systemic sclerosis
Date of designation: 15/05/2009, Positive, Last updated: 13/02/2023 -
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Orphan designation: Treprostinil sodium for: Treatment of idiopathic pulmonary fibrosis
Date of designation: 16/03/2022, Positive, Last updated: 13/02/2023 -
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Orphan designation: Ralinepag for: Treatment of pulmonary arterial hypertension
Date of designation: 11/01/2019, Positive, Last updated: 13/02/2023 -
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Orphan designation: Fenfluramine hydrochloride for: Treatment of Lennox-Gastaut syndrome
Date of designation: 27/02/2017, Positive, Last updated: 07/02/2023