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2797 results
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Orphan designation: Dabrafenib mesylate for: Treatment of glioma (updated)
Date of designation: 09/12/2020, Positive, Last updated: 15/09/2023 -
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Orphan designation: Asunercept for: Treatment of myelodysplastic syndromes (updated)
Date of designation: 23/08/2017, Positive, Last updated: 15/09/2023 -
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Orphan designation: Recombinant fusion protein consisting of the extracellular portion of CD95 fused to the Fc part of a human IgG1 molecule for: Treatment of glioma (updated)
Date of designation: 28/01/2010, Positive, Last updated: 15/09/2023 -
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Orphan designation: Recombinant fusion protein consisting of the extracellular portion of CD95 fused to the Fc part of a human IgG1 molecule for: Prevention of graft-versus-host disease (updated)
Date of designation: 31/10/2006, Positive, Last updated: 15/09/2023 -
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Orphan designation: 2-[6-(6,7-Dimethoxyquinolin-3-yl)pyridin-3-yl]-N-[3-(1,1,1-trifluoro-2-methylpropan-2-yl)-1,2-oxazol-5-yl]acetamide (zeteletinib) for: Treatment of medullary thyroid carcinoma (updated)
Date of designation: 19/02/2021, Positive, Last updated: 13/09/2023 -
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Orphan designation: Entinostat for: Treatment of Hodgkin's lymphoma (updated)
Date of designation: 10/06/2010, Positive, Last updated: 13/09/2023 -
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Orphan designation: Mixture of seven synthetic fragments consisting of p21 RAS peptides for: Treatment of pancreatic cancer (updated)
Date of designation: 05/08/2011, Positive, Last updated: 13/09/2023 -
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Orphan designation: Pomalidomide for: Treatment of multiple myeloma (updated)
Date of designation: 08/10/2009, Expired, Last updated: 13/09/2023 -
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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Date of designation: 20/03/2007, Withdrawn, Last updated: 12/09/2023 -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Date of designation: 26/02/2019, Withdrawn, Last updated: 12/09/2023 -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: Lentiviral vector carrying the Fanconi anaemia-A (FANCA) gene for: Treatment of Fanconi anaemia type A (updated)
Date of designation: 17/12/2010, Positive, Last updated: 11/09/2023 -
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Orphan designation: 2',3',5'-Tri-O-acetyluridine for: Treatment of 5-fluorouracil overdose (updated)
Date of designation: 15/05/2009, Positive, Last updated: 11/09/2023 -
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Orphan designation: 4-Amino-(6R,S)-5,6,7,8-tetrahydro-L-biopterin dihydrochloride (ronopterin) for: Treatment of moderate and severe traumatic brain injury
Date of designation: 28/08/2006, Positive, Last updated: 11/08/2023 -
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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: Recombinant human factor IX protein modified with three point mutations (dalcinonacog alfa) for: Treatment of haemophilia B
Date of designation: 20/06/2017, Positive, Last updated: 03/08/2023 -
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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: Ganaxolone for: Treatment of CDKL5 deficiency disorder
Date of designation: 13/11/2019, Positive, Last updated: 31/07/2023 -
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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: Tobramycin for: Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis
Date of designation: 17/03/2003, Expired, 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