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2394 results
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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: Codon-optimised human ornithine transcarbamylase mRNA complexed with lipid-based nanoparticles for: Treatment of ornithine transcarbamylase deficiency (updated)
Date of first decision: 27/06/2018, Withdrawn, Last updated: 28/04/2021 -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: tripotassium citrate monohydrate, Potassium hydrogen carbonate for: Treatment of distal renal tubular acidosis (updated)
Date of first decision: 20/06/2017, Positive, Last updated: 22/04/2021 -
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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: Ixazomib for: Treatment of systemic light chain amyloidosis (updated)
Date of first decision: 08/11/2012, Withdrawn, Last updated: 09/04/2021 -
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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: 2-((2-ethyl-6-(4-(2-(3-hydroxyazetidin-1-yl)-2-oxoethyl)-piperazin-1-yl)-8-methylimidazo[1,2-alpha]pyridin-3-yl)-(methyl)amino)-4-(4-fluorophenyl)-thiazole-5-carbonitrile (ziritaxestat) for: Treatment of idiopathic pulmonary fibrosis (updated)
Date of first decision: 29/08/2016, Withdrawn, Last updated: 09/04/2021 -
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Orphan designation: Chimeric monoclonal antibody against claudin 6 for: Treatment of ovarian cancer (updated)
Date of first decision: 24/01/2013, Withdrawn, Last updated: 08/04/2021 -
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Orphan designation: Recombinant human glutamate oxaloacetate transaminase 1 for: Treatment of glioma (updated)
Date of first decision: 12/02/2015, Withdrawn, Last updated: 07/04/2021 -
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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: romilkimab for: Treatment of systemic sclerosis (updated)
Date of first decision: 09/01/2020, Withdrawn, Last updated: 06/04/2021 -
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Orphan designation: ziritaxestat for: Treatment of systemic sclerosis (updated)
Date of first decision: 09/01/2020, Withdrawn, Last updated: 06/04/2021 -
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Orphan designation: Adeno-associated virus serotype 9 containing the human ASPA gene for: Treatment of Canavan disease (updated)
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 (updated)
Date of first decision: 09/01/2020, Positive, Last updated: 06/04/2021