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4183 results
Medicine
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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Date of first decision: 08/11/2012, Withdrawn, Last updated: 09/04/2021 -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Date of first decision: 29/08/2016, Withdrawn, Last updated: 09/04/2021 -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Date of first decision: 24/01/2013, Withdrawn, Last updated: 08/04/2021 -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Date of first decision: 12/02/2015, Withdrawn, Last updated: 07/04/2021 -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 -
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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: Terguride for: Treatment of systemic sclerosis (updated)
Date of first decision: 24/01/2013, Withdrawn, 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: Pirfenidone for: Treatment of idiopathic pulmonary fibrosis (updated)
Date of first decision: 16/11/2004, Expired, Last updated: 22/03/2021 -
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Orphan designation: milciclib maleate for: Treatment of malignant thymoma (updated)
Date of first decision: 08/11/2012, Withdrawn, Last updated: 22/03/2021 -
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Orphan designation: Allogeneic umbilical cord blood CD34+ cells cultured ex vivo with Notch ligand Delta1 (dilanubicel) for: Treatment in haematopoietic stem cell transplantation (updated)
Date of first decision: 17/01/2018, Withdrawn, 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