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2626 results
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Orphan designation: N-({Carbamoylmethyl-[3-(2-oxo-pyrrolidin-1-yl)-propyl]-carbamoyl}-methyl)-2-[2-(2-fluoro-phenyl)-ethylamino]-N-isobutyl-acetamide for: Treatment of optic neuritis (updated)
Date of designation: 31/03/2014, Positive, Last updated: 05/07/2022 -
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Orphan designation: Recombinant human acid sphingomyelinase (also known as olipudase alfa) for: Treatment of Niemann-Pick disease (updated)
Date of designation: 05/12/2016, Positive, Last updated: 05/07/2022 -
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Orphan designation: N-Hydroxy-4-(3-methyl-2-(S)-phenyl-butyrylamino) benzamide for: Treatment of neurofibromatosis type 2 (updated)
Date of designation: 26/04/2012, Positive, Last updated: 05/07/2022 -
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Orphan designation: dry extract from birch bark (DER 5-10 : 1), extraction solvent n-heptane 95% (w/w) (birch bark extract) for: Treatment of epidermolysis bullosa (updated)
Date of designation: 23/02/2011, Positive, Last updated: 04/07/2022 -
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Orphan designation: (2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amNide for: Treatment of microscopic polyangiitis (avacopan) (updated)
Date of designation: 19/11/2014, Positive, Last updated: 04/07/2022 -
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Orphan designation: (2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amide (avacopan) for: Treatment of granulomatosis with polyangiitis (updated)
Date of designation: 19/11/2014, Positive, Last updated: 04/07/2022 -
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Orphan designation: Human autologous mesenchymal adult stem cells extracted from adipose tissue for: Treatment of anal fistula (updated)
Date of designation: 26/08/2005, Withdrawn, Last updated: 01/07/2022 -
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Orphan designation: Setmelanotide for: Treatment of Alström syndrome (updated)
Date of designation: 09/01/2020, Positive, Last updated: 01/07/2022 -
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Orphan designation: Setmelanotide for: Treatment of Bardet-Biedl syndrome (updated)
Date of designation: 21/08/2019, Positive, Last updated: 01/07/2022 -
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Orphan designation: Setmelanotide for: Treatment of leptin receptor deficiency (updated)
Date of designation: 19/11/2018, Positive, Last updated: 01/07/2022 -
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Orphan designation: Setmelanotide for: Treatment of pro-opiomelanocortin deficiency (updated)
Date of designation: 14/07/2016, Positive, Last updated: 01/07/2022 -
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Orphan designation: Setmelanotide for: Treatment of Prader-Willi syndrome (updated)
Date of designation: 27/06/2016, Positive, Last updated: 01/07/2022 -
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Orphan designation: Rezafungin acetate for: Treatment of invasive candidiasis (updated)
Date of designation: 06/01/2021, Positive, Last updated: 30/06/2022 -
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Orphan designation: Recombinant human monoclonal antibody to insulin receptor for: Treatment of congenital hyperinsulinism (updated)
Date of designation: 14/07/2016, Positive, Last updated: 30/06/2022 -
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Orphan designation: 2-methyl-1-[(4-[6-(trifluoromethyl)pyridin-2-yl]-6-{[2-(trifluoromethyl)pyridin-4-yl]amino}-1,3,5-triazin-2-yl)amino]propan-2-ol methanesulfonate (enasidenib) for: Treatment of acute myeloid leukaemia (updated)
Date of designation: 28/04/2016, Positive, Last updated: 29/06/2022 -
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Orphan designation: Mosunetuzumab for: Treatment of follicular lymphoma (updated)
Date of designation: 12/11/2021, Positive, Last updated: 23/06/2022 -
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Orphan designation: Chimeric locked nucleic acid deoxynucleoside phosphorothioate-linked oligonucleotide inhibitor directed against microRNA-155-5p for: Treatment of cutaneous T-cell lymphoma (updated)
Date of designation: 22/05/2017, Withdrawn, Last updated: 22/06/2022 -
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Orphan designation: Protein-based delivery vector carrying a DNA payload encoding an RNA-guided nuclease that targets stx genes of Shiga toxin-producing Escherichia coli for: Prevention of haemolytic uraemic syndrome (updated)
Date of designation: 21/08/2020, Positive, Last updated: 22/06/2022 -
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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 (updated)
Date of designation: 24/04/2019, Positive, Last updated: 22/06/2022 -
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Orphan designation: Modified mRNA encoding human methylmalonyl-coenzyme A mutase encapsulated into lipid nanoparticles for: Treatment of methylmalonic acidaemia (updated)
Date of designation: 25/05/2018, Positive, Last updated: 22/06/2022 -
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Orphan designation: Synthetic signal peptide of human mucin-1 (amino acids 1-21) for: Treatment of plasma cell myeloma (updated)
Date of designation: 15/01/2015, Positive, Last updated: 22/06/2022 -
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Orphan designation: Methyl 4,6-diamino-2-[1-(2-fluorobenzyl)-1H-pyrazolo[3,4-b]pyridine-3-yl]-5-pyrimidinyl(methyl)carbamate (riociguat) for: Treatment of pulmonary arterial hypertension (updated)
Date of designation: 20/12/2007, Withdrawn, Last updated: 14/06/2022 -
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Orphan designation: (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride (maralixibat chloride) for: Treatment of primary sclerosing cholangitis (updated)
Date of designation: 16/01/2014, Withdrawn, Last updated: 14/06/2022 -
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Orphan designation: (4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5- tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride (maralixibat chloride) for: Treatment of primary biliary cirrhosis (updated)
Date of designation: 16/01/2014, Withdrawn, Last updated: 14/06/2022 -
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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 (updated)
Date of designation: 01/04/2019, Positive, Last updated: 14/06/2022