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2782 results
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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Date of designation: 16/03/2022, Withdrawn, Last updated: 08/08/2023 -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Date of designation: 14/12/2018, Withdrawn, Last updated: 08/08/2023 -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Date of designation: 25/07/2006, Withdrawn, Last updated: 08/08/2023 -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Date of designation: 15/10/2021, Withdrawn, Last updated: 31/07/2023 -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Date of designation: 23/08/2017, Withdrawn, Last updated: 27/07/2023 -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 -
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Orphan designation: Mavorixafor for: Treatment of WHIM syndrome
Date of designation: 25/07/2019, Positive, Last updated: 26/07/2023 -
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Orphan designation: Iodine (124I) evuzamitide for: Diagnosis of ATTR amyloidosis
Date of designation: 09/12/2022, Positive, Last updated: 25/07/2023 -
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Orphan designation: Iodine (124I) evuzamitide for: Diagnosis of AL amyloidosis
Date of designation: 11/11/2022, Positive, Last updated: 25/07/2023 -
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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: Dersimelagon for: Treatment of erythropoietic protoporphyria
Date of designation: 16/03/2022, Positive, Last updated: 21/07/2023 -
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Orphan designation: edaravone for: Treatment of amyotrophic lateral sclerosis
Date of designation: 19/06/2015, Positive, Last updated: 21/07/2023 -
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Orphan designation: Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib) for: Treatment of acute lymphoblastic leukaemia
Date of designation: 02/02/2010, Expired, Last updated: 20/07/2023 -
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Orphan designation: Benzamide, 3-(2-imidazo[1,2-b]pyridazin-3-ylethynyl)-4-methyl-N-[4-[(4-methyl-1-piperazinyl)methyl]-3-(trifluoromethyl)phenyl] (ponatinib) for: Treatment of chronic myeloid leukaemia
Date of designation: 02/02/2010, Expired, Last updated: 20/07/2023 -
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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: 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: Glofitamab for: Treatment of diffuse large B-cell lymphoma
Date of designation: 15/10/2021, Positive, Last updated: 18/07/2023 -
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Orphan designation: Recombinant human acid sphingomyelinase (also known as olipudase alfa) for: Treatment of Niemann-Pick disease
Date of designation: 24/06/2022, Positive, Last updated: 18/07/2023 -
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Orphan designation: (1R,2R)-octanoic acid[2-(2',3'-dihydro-benzo[1,4] dioxin-6'-yl)-2-hydroxy-1-pyrrolidin-1-ylmethyl-ethyl]-amide-L-tartaric acid salt (eliglustat) for: Treatment of Gaucher disease
Date of designation: 04/12/2007, Positive, Last updated: 18/07/2023 -
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against antithrombin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues (fitusiran) for: Treatment of haemophilia A
Date of designation: 29/07/2014, Positive, Last updated: 18/07/2023