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2394 results
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Orphan designation: Tadekinig alfa for: Treatment of haemophagocytic lymphohistiocytosis (updated)
Date of first decision: 14/10/2016, Positive, Last updated: 19/02/2021 -
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Orphan designation: Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain (luspatercept) for: Treatment of beta thalassaemia intermedia and major (updated)
Date of first decision: 29/07/2014, Positive, Last updated: 18/02/2021 -
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Orphan designation: Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain (luspatercept) for: Treatment of myelodysplastic syndromes (updated)
Date of first decision: 22/08/2014, Positive, Last updated: 18/02/2021 -
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Orphan designation: Luspatercept for: Treatment of myelofibrosis (updated)
Date of first decision: 28/02/2020, Positive, Last updated: 18/02/2021 -
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Orphan designation: 5-(Ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole (ezutromid) for: Treatment of Duchenne muscular dystrophy (updated)
Date of first decision: 04/12/2008, Withdrawn, Last updated: 10/02/2021 -
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Orphan designation: human anti-promyostatin monoclonal antibody (apitegromab) for: Treatment of spinal muscular atrophy (updated)
Date of first decision: 14/12/2018, Positive, Last updated: 09/02/2021 -
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Orphan designation: Autologous CD4+ and CD8+ T cells expressing a CD19-specific chimeric antigen receptor (lisocabtagene maraleucel) for: Treatment of diffuse large B-cell lymphoma (updated)
Date of first decision: 17/07/2017, Positive, Last updated: 09/02/2021 -
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Orphan designation: Autologous CD4+ and CD8+ T cells expressing a CD19-specific chimeric antigen receptor (lisocabtagene maraleucel) for: Treatment of follicular lymphoma (updated)
Date of first decision: 25/05/2018, Positive, Last updated: 09/02/2021 -
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Orphan designation: Lisocabtagene maraleucel for: Treatment of primary mediastinal large B-cell lymphoma (updated)
Date of first decision: 19/11/2018, Positive, Last updated: 09/02/2021 -
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Orphan designation: Human monoclonal antibody against Pseudomonas aeruginosa IATS-O1 for: Treatment of pneumonia caused by serotype O1 Pseudomonas aeruginosa (updated)
Date of first decision: 28/01/2010, Withdrawn, Last updated: 03/02/2021 -
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Orphan designation: RNA, [P-deoxy-P-(dimethylamino)] (2',3'-dideoxy-2',3'-imino-2',3'-seco) (2'a→5') (C-m5U-m5U-A-C-A-G-G-C-m5U-C-C-A-A-m5U-A-G-m5U-G-G-m5U-C-A-G-m5U), 5' [P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylaminophosphonamidate], 3'-[2'a-[N2-acetyl-L-arginyl-6-aminohexanoyl-L-arginyl-L-arginyl-β-alanyl-L-arginyl-L-arginyl-6-aminohexanoyl-L-arginyl-L-arginyl-β-alanyl-L-arginyl-6-aminohexanoyl-β-alanyl], octahydrochloride for: Treatment of Duchenne muscular dystrophy (updated)
Date of first decision: 02/02/2010, Withdrawn, Last updated: 03/02/2021 -
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Orphan designation: Sodium thiosulfate for: Treatment of calciphylaxis (updated)
Date of first decision: 23/02/2011, Withdrawn, Last updated: 03/02/2021 -
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Orphan designation: Adeno-associated viral vector serotype 8 containing the human UGT1A1 gene (volrubigene ralaparvovec) for: Treatment of Crigler-Najjar syndrome (updated)
Date of first decision: 18/11/2016, Withdrawn, Last updated: 03/02/2021 -
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Orphan designation: Heparin-activated recombinant human fibroblast growth factor 1 (on a biodegradable device made from alpha-calcium sulfate hemihydrate) for: Treatment of traumatic spinal cord injury (updated)
Date of first decision: 27/07/2010, Withdrawn, Last updated: 03/02/2021 -
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Orphan designation: Poly(oxy-1,2-ethanediyl), alpha-hydro-omega-hydroxy-,15,15'-diester with N-acetyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-alpha-aspartyl-L-tryptophylglycyl-L-alanyl-L-histidyl-L-arginyl-L-cysteinyl-L-threonyl-2-[2-(2-aminoethoxy)ethoxy]acetyl-N6-carboxy-L-lysinamide cyclic (2.fwdarw.12)-(disulfide); where two identical synthetic peptide domains are covalently linked at the ends of the polyethylene glycol chain (pegcetacoplan) for: Treatment of C3 glomerulopathy (updated)
Date of first decision: 21/08/2019, Positive, Last updated: 03/02/2021 -
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Orphan designation: 2-isopropyl-3H-naphtho[1,2-d]imidazole-4,5-dione for: Treatment of mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (updated)
Date of first decision: 12/12/2017, Positive, Last updated: 03/02/2021 -
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Orphan designation: ciclosporin for: Treatment of moderate and severe closed traumatic brain injury (updated)
Date of first decision: 01/10/2010, Positive, Last updated: 03/02/2021 -
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Orphan designation: 2-Hydroxyoleic acid for: Treatment of glioma (updated)
Date of first decision: 27/10/2011, Positive, Last updated: 03/02/2021 -
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Orphan designation: (1S,3S)-3-amino-4-(difluoromethylene) cyclopentanecarboxylic acid hydrochloride for: Treatment of West syndrome (updated)
Date of first decision: 09/02/2012, Withdrawn, Last updated: 03/02/2021 -
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Orphan designation: Rovalpituzumab tesirine for: Treatment of small cell lung cancer (updated)
Date of first decision: 30/05/2016, Withdrawn, Last updated: 03/02/2021 -
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Orphan designation: Oxalobacter formigenes strain HC-1 for: Treatment of primary hyperoxaluria (updated)
Date of first decision: 17/02/2006, Positive, Last updated: 02/02/2021 -
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Orphan designation: Oxalobacter formigenes strain HC-1 for: Treatment of short bowel syndrome (updated)
Date of first decision: 15/10/2014, Positive, Last updated: 02/02/2021 -
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Orphan designation: Somapacitan for: Treatment of growth hormone deficiency (updated)
Date of first decision: 24/08/2018, Positive, Last updated: 01/02/2021 -
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Orphan designation: pemigatinib for: Treatment of myeloid/lymphoid neoplasms with eosinophilia and rearrangement of PDGFRA, PDGFRB, or FGFR1, or with PCM1-JAK (updated)
Date of first decision: 17/10/2019, Positive, Last updated: 01/02/2021 -
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Orphan designation: Clofazimine for: Treatment of nontuberculous mycobacterial lung disease
Date of first decision: 21/08/2019, Positive, Last updated: 28/01/2021