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732 results
Medicine
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Orphan designation: Enzastaurin hydrochloride for: Treatment of diffuse large B-cell lymphoma (updated)
Date of designation: 20/03/2007, Withdrawn, Last updated: 12/09/2023 -
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Orphan designation: Risdiplam for: Treatment of spinal muscular atrophy (updated)
Date of designation: 26/02/2019, Withdrawn, Last updated: 12/09/2023 -
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Orphan designation: Adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human iduronate 2-sulfatase gene for: Treatment of mucopolysaccharidosis type II (Hunter's syndrome) (updated)
Date of designation: 17/01/2018, Withdrawn, Last updated: 12/09/2023 -
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Orphan designation: Adeno-associated viral vector serotype 2/6 encoding zinc-finger nucleases and the human alpha L-iduronidase gene (devafidugene civaparvovec) for: Treatment of mucopolysaccharidosis type I (updated)
Date of designation: 17/01/2018, Withdrawn, Last updated: 12/09/2023 -
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Orphan designation: Adeno-associated virus serotype HSC 15 expressing human iduronate 2-sulfatase (Bevufenogene nofeparvovec) for: Treatment of mucopolysaccharidosis type II (Hunter Syndrome)
Date of designation: 16/03/2022, Withdrawn, Last updated: 08/08/2023 -
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Orphan designation: Adeno-associated virus serotype HSC15 expressing human phenylalanine hydroxylase for: Treatment of phenylalanine hydroxylase deficiency
Date of designation: 14/12/2018, Withdrawn, Last updated: 08/08/2023 -
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Orphan designation: Human telomerase reverse transcriptase peptide (611-626) (tertomotide) for: Treatment of pancreatic cancer
Date of designation: 25/07/2006, Withdrawn, Last updated: 08/08/2023 -
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Orphan designation: Humanised IgG1 monoclonal antibody against SEZ6 linked to N-acetyl-calicheamicin for: Treatment of small cell lung cancer
Date of designation: 15/10/2021, Withdrawn, Last updated: 31/07/2023 -
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Orphan designation: Itraconazole for: Treatment of naevoid basal-cell carcinoma syndrome (Gorlin syndrome)
Date of designation: 23/08/2017, Withdrawn, Last updated: 27/07/2023 -
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Orphan designation: Fusion protein composed of the first 2 immunoglobulin-like domains of the human Roundabout guidance receptor 2 fused to a human IgG1 crystallised fragment for: Treatment of focal segmental glomerulosclerosis
Date of designation: 16/05/2022, Withdrawn, Last updated: 27/07/2023 -
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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: Adeno-associated virus serotype HSC15 expressing human arylsulfatase A gene for: treatment of metachromatic leukodystrophy
Date of designation: 26/06/2020, Withdrawn, Last updated: 21/07/2023 -
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Orphan designation: Human embryonic stem-cell-derived retinal pigment epithelial cells for: Treatment of Stargardt's disease
Date of designation: 21/06/2011, Withdrawn, Last updated: 19/07/2023 -
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Orphan designation: Cedazuridine, decitabine for: Treatment of acute myeloid leukaemia
Date of designation: 10/12/2021, Withdrawn, Last updated: 06/07/2023 -
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Orphan designation: Modified mRNA encoding human methylmalonyl-coenzyme A mutase encapsulated into lipid nanoparticles for: Treatment of methylmalonic acidaemia
Date of designation: 25/05/2018, Withdrawn, Last updated: 26/06/2023 -
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Orphan designation: Recombinant self-complementary adeno-associated viral vector serotype 9 containing the human CLN3 gene for: Treatment of neuronal ceroid lipofuscinosis
Date of designation: 08/10/2009, Withdrawn, Last updated: 20/06/2023 -
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Orphan designation: Doxorubicin(6-maleimidocaproyl)hydrazone (aldoxorubicin) for: Treatment of soft tissue sarcoma
Date of designation: 26/03/2014, Withdrawn, Last updated: 06/06/2023 -
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Orphan designation: 5'-ASCSASTSCSASGSTSCSTSGSASUSASASGSCSTSA-3' (lademirsen) for: Treatment of Alport syndrome
Date of designation: 19/03/2015, Withdrawn, Last updated: 05/06/2023 -
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Orphan designation: Recombinant human acid alpha-glucosidase for: Treatment of glycogen storage disease type II (Pompe's disease)
Date of designation: 21/03/2018, Withdrawn, Last updated: 30/05/2023 -
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Orphan designation: Pegunigalsidase alfa for: Treatment of Fabry disease
Date of designation: 12/12/2017, Withdrawn, Last updated: 08/05/2023 -
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Orphan designation: Rozanolixizumab for: Treatment of immune thrombocytopenia
Date of designation: 11/01/2019, Withdrawn, Last updated: 08/05/2023 -
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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: (R)-1-[1-(4-acetoxy-3,3-dimethyl-2-oxo-butyl)-2-oxo-5-(pyridin-2-yl)-2,3-dihydro-1H-benzo[e][1,4]diazepin-3-yl]-3-(3-methylamino-phenyl)-urea (ceclazepide) for: Treatment of gastro-entero-pancreatic neuroendocrine tumours
Date of designation: 14/12/2015, Withdrawn, Last updated: 04/04/2023 -
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Orphan designation: Soluble recombinant human fibroblast growth factor receptor 3 for: Treatment of achondroplasia
Date of designation: 27/02/2017, Withdrawn, Last updated: 24/03/2023 -
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Orphan designation: miglustat for: Treatment of glycogen storage disease type II (Pompe's disease)
Date of designation: 11/01/2019, Withdrawn, Last updated: 24/03/2023