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12 results
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Orphan designation: Marzeptacog alfa (activated) for: Treatment of haemophilia B
Date of designation: 01/04/2019, Positive, Last updated: 03/08/2023 -
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Orphan designation: Codon-optimised human cystic fibrosis transmembrane conductance regulator messenger ribonucleic acid complexed with lipid-based nanoparticles, pomulmeran for: Treatment of cystic fibrosis
Date of designation: 01/04/2019, Withdrawn, Last updated: 21/07/2023 -
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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
Date of designation: 01/04/2019, Positive, Last updated: 18/07/2023 -
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Orphan designation: 3-(3-(3,5-bis(trifluoromethyl)phenyl)-1h-pyrazol-1-yl)propanoic acid for: Treatment of Stargardt's disease
Date of designation: 24/04/2019, Withdrawn, Last updated: 13/04/2023 -
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Orphan designation: Modified messenger ribonucleic acid encoding human propionyl-coenzyme A carboxylase alpha and beta subunits encapsulated into lipid nanoparticle for: Treatment of propionic acidaemia
Date of designation: 24/04/2019, Positive, Last updated: 22/06/2022 -
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Orphan designation: Adeno-associated viral vector serotype rh10 containing the human cholesterol 24-hydroxylase gene for: Treatment of Huntington’s disease
Date of designation: 01/04/2019, Positive, Last updated: 14/06/2022 -
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Orphan designation: Human culture expanded autologous mesenchymal stromal cells for: Treatment of amyotrophic lateral sclerosis
Date of designation: 24/04/2019, Positive, Last updated: 03/05/2022 -
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Orphan designation: Balipodect for: Treatment of fragile X syndrome
Date of designation: 14/04/2019, Positive, Last updated: 26/08/2020 -
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Orphan designation: 4-hydroxy-6-{2-[4-(trifluoromethyl)phenyl]ethyl}pyridazin-3(2H)-one for: Treatment of Friedreich’s ataxia
Date of designation: 01/04/2019, Withdrawn, Last updated: 20/11/2019 -
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Orphan designation: 2-[3-(2-chloro-4-{[5-cyclopropyl-3-(2,6-dichlorophenyl)-1,2-oxazol-4-yl]methoxy}phenyl)-3-hydroxyazetidin-1-yl]pyridine-4-carboxylic acid-2-amino-2-(hydroxymethyl)propane-1,3-diol (1/1) for: Treatment of primary sclerosing cholangitis
Date of designation: 01/04/2019, Positive, Last updated: 07/05/2019 -
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Orphan designation: Autologous human bone marrow-derived haematopoietic and mesenchymal stem cells depleted of erythrocytes, monocytes and lymphocytes for: Treatment of spinal cord injury
Date of designation: 24/04/2019, Positive, Last updated: 14/06/2019 -
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Orphan designation: Sodium benzoate, Sodium phenylacetate for: Treatment of ornithine transcarbamylase deficiency
Date of designation: 24/04/2019, Positive, Last updated: 14/06/2019