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1723 results
Medicine
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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 (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 -
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Orphan designation: Sparsentan for: Treatment of primary IgA nephropathy (updated)
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 (updated)
Date of first decision: 11/11/2015, Positive, Last updated: 24/03/2021 -
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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: 22/03/2021 -
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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 (updated)
Date of first decision: 23/08/2017, Positive, Last updated: 22/03/2021 -
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Orphan designation: (E)-2,4,6-trimethoxystyryl-3-carboxymethylamino-4-methoxybenzyl-sulfone sodium salt for: Treatment of myelodysplastic syndromes (updated)
Date of first decision: 26/04/2012, Positive, Last updated: 22/03/2021 -
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Orphan designation: Genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor for: Treatment of malignant mesothelioma (updated)
Date of first decision: 16/12/2014, Positive, Last updated: 19/03/2021 -
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Orphan designation: genetically modified serotype 5/3 adenovirus coding for granulocyte-macrophage colony-stimulating factor for: Treatment of soft tissue sarcoma (updated)
Date of first decision: 19/06/2013, Positive, Last updated: 19/03/2021 -
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Orphan designation: Genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor for the treatment of ovarian cancer for: Treatment of ovarian cancer (updated)
Date of first decision: 29/04/2014, Positive, Last updated: 19/03/2021 -
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Orphan designation: dry extract from birch bark (DER 5-10 : 1), extraction solvent n-heptane 95% (w/w) for: Treatment of epidermolysis bullosa
Date of first decision: 23/02/2011, Positive, Last updated: 09/03/2021 -
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Orphan designation: Murine anti-CD22 antibody variable region fused to truncated Pseudomonas exotoxin 38 ( Moxetumomab pasudotox) for: Treatment of hairy cell leukaemia
Date of first decision: 05/12/2008, Positive, Last updated: 09/03/2021 -
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Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of post-polycythaemia vera myelofibrosis
Date of first decision: 26/11/2010, Positive, Last updated: 03/03/2021 -
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Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of post-essential thrombocythaemia myelofibrosis
Date of first decision: 26/11/2010, Positive, Last updated: 03/03/2021 -
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Orphan designation: N-tert-butyl-3-[(5-methyl-2-{[4-(2-pyrrolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl)amino] benzenesulfonamide dihydrochloride monohydrate (fedratinib) for: Treatment of primary myelofibrosis
Date of first decision: 01/10/2010, Positive, Last updated: 03/03/2021 -
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Orphan designation: (R)-1-[1-(4-acetoxy-3,3-dimethyl-2-oxo-butyl)-2-oxo-5-(pyridin-2-yl)-2,3-dihydro-1H-benzo[e][1,4]diazepin-3-yl]-3-(3-methylamino-phenyl)-urea (ceclazepide) for: Treatment of gastro-entero-pancreatic neuroendocrine tumours
Date of first decision: 14/12/2015, Positive, Last updated: 02/03/2021 -
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Orphan designation: R-1-[2,3-dihydro-2-oxo-1-pivaloylmethyl-5-(2-pyridyl)-1 H-1,4-benzodiazepin-3-yl]-3-(3-methylaminophenyl)urea (netazepide) for: Treatment of gastric carcinoid
Date of first decision: 14/06/2007, Positive, Last updated: 02/03/2021 -
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Orphan designation: N-(4-(1-cyanocyclopentyl)phenyl)-2-(4-pyridinylmethyl)amino-3-pyridinecarboxamide methanesulfonate for: Treatment of gastric cancer
Date of first decision: 27/02/2017, Positive, Last updated: 02/03/2021 -
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Orphan designation: N-acetylgalactosamine-conjugated synthetic double-stranded oligomer specific to serpin family A member 1 gene for: Treatment of congenital alpha-1 antitrypsin deficiency
Date of first decision: 31/07/2018, Positive, Last updated: 02/03/2021