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1778 results
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Orphan designation: Ivacaftor, N-(1,3-dimethyl-1H-pyrazole-4-sulfonyl)-6-[3-(3,3,3-trifluoro-2,2-dimethylpropoxy)-1H-pyrazol-1-yl]-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carboxamide, tezacaftor for: Treatment of cystic fibrosis (updated)
Date of first decision: 14/12/2018, Positive, Last updated: 06/05/2021 -
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Orphan designation: Teicoplanin for: Treatment of cystic fibrosis (updated)
Date of first decision: 23/08/2017, Positive, Last updated: 06/05/2021 -
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Orphan designation: Anti-neonatal Fc receptor human monoclonal antibody for: Prevention of haemolytic disease of the foetus and newborn (updated)
Date of first decision: 17/10/2019, Positive, Last updated: 05/05/2021 -
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Orphan designation: Risdiplam for: Treatment of spinal muscular atrophy (updated)
Date of first decision: 26/02/2019, Positive, Last updated: 04/05/2021 -
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Orphan designation: pemigatinib for: Treatment of biliary tract cancer (updated)
Date of first decision: 24/08/2018, Positive, Last updated: 04/05/2021 -
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Orphan designation: L-cystine bis(N-methylpiperazide) for: Treatment of cystinuria (updated)
Date of first decision: 27/06/2018, Positive, Last updated: 30/04/2021 -
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Orphan designation: Mexiletine hydrochloride for: Treatment of non-dystrophic myotonia (updated)
Date of first decision: 07/06/2013, Positive, Last updated: 30/04/2021 -
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Orphan designation: lonapegsomatropin for: Treatment of growth hormone deficiency (updated)
Date of first decision: 17/10/2019, Positive, Last updated: 29/04/2021 -
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Orphan designation: 1,2:5,6-Dianhydrogalactitol for: Treatment of glioma (updated)
Date of first decision: 24/01/2013, Positive, Last updated: 28/04/2021 -
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Orphan designation: Cannabidiol for: Treatment of tuberous sclerosis (updated)
Date of first decision: 17/01/2018, Positive, Last updated: 26/04/2021 -
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Orphan designation: Diclofenamide for: Treatment of periodic paralysis (updated)
Date of first decision: 17/02/2016, Positive, Last updated: 26/04/2021 -
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Orphan designation: (3'R,4'S,5'R)-N-[(3R,6S)-6-carbamoyltetrahydro-2H-pyran-3-yl]-6''-chloro-4'-(2-chloro-3-fluoropyridin-4-yl)-4,4-dimethyl-2''-oxo-1'',2''-dihydrodispiro[cyclohexane-1,2'-pyrrolidine-3',3''-indole]-5'-carboxamide mono(4-methylbenzenesulfonate) monohydrate for: Treatment of soft tissue sarcoma (updated)
Date of first decision: 20/03/2017, Positive, Last updated: 22/04/2021 -
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Orphan designation: Eicosapentaenoic acid for: Treatment of familial adenomatous polyposis (FAP) (updated)
Date of first decision: 08/10/2009, Positive, Last updated: 22/04/2021 -
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Orphan designation: Somapacitan for: Treatment of growth hormone deficiency (updated)
Date of first decision: 24/08/2018, Positive, Last updated: 15/04/2021 -
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Orphan designation: Ribavirin for: Treatment of Lassa fever (updated)
Date of first decision: 21/02/2018, Positive, Last updated: 12/04/2021 -
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Orphan designation: Ribavirin for: Treatment of Crimean-Congo haemorrhagic fever (updated)
Date of first decision: 21/03/2018, Positive, Last updated: 12/04/2021 -
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Orphan designation: Adeno-associated viral vector serotype 9 containing the human cardiac calsequestrin gene for: Treatment of catecholaminergic polymorphic ventricular tachycardia (updated)
Date of first decision: 29/07/2014, Positive, Last updated: 09/04/2021 -
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Orphan designation: Radio-iodinated (131I) anti-CD45 murine monoclonal antibody for: Treatment in haematopoietic stem cell transplantation (updated)
Date of first decision: 14/10/2016, Positive, Last updated: 09/04/2021 -
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Orphan designation: Allogeneic multi-virus specific T lymphocytes targeting BK virus, cytomegalovirus, human herpesvirus-6, Epstein Barr virus and adenovirus for: Treatment in haematopoietic stem cell transplantation (updated)
Date of first decision: 24/03/2020, Positive, Last updated: 07/04/2021 -
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Orphan designation: Synthetic 12 amino acid peptide designed after subcommissural organ-spondin for: Treatment of spinal cord injury (updated)
Date of first decision: 13/11/2013, Positive, Last updated: 07/04/2021 -
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Orphan designation: Adeno-associated virus serotype 9 containing the human ASPA gene for: Treatment of Canavan disease
Date of first decision: 04/06/2020, Positive, Last updated: 06/04/2021 -
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Orphan designation: tripotassium citrate monohydrate, Potassium hydrogen carbonate for: Treatment of cystinuria
Date of first decision: 09/01/2020, Positive, Last updated: 06/04/2021 -
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Orphan designation: Sparsentan for: Treatment of primary IgA nephropathy
Date of first decision: 19/10/2020, Positive, Last updated: 24/03/2021 -
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Orphan designation: 4'-[(2-butyl-4-oxo-1,3-diazaspiro[4.4]non-1-en-3-yl)methyl]-N-(4,5-dimethyl-3-isoxazolyl)-2'-(ethoxymethyl)-[1,1'-biphenyl]-2-sulfonamide for: Treatment of focal segmental glomerulosclerosis
Date of first decision: 11/11/2015, Positive, Last updated: 24/03/2021 -
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Orphan designation: Salmonella typhi Ty21a strain transfected with a plasmid vector encoding the human vascular endothelial growth factor receptor 2 for: Treatment of glioma
Date of first decision: 23/08/2017, Positive, Last updated: 22/03/2021