2361 results
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Orphan designation: Sodium (2R,3S,5R)-5-(4-amino-2-oxo-1,3,5-triazin-1(2H)-yl)-2-(hydroxymethyl)tetrahydrofuran-3-yl ((2R,3S,5R)-5-(2-amino-6-oxo-1H-purin-9(6H)-yl)-3-hydroxytetrahydrofuran-2-yl)methyl phosphate (guadecitabine) for: Treatment of acute myeloid leukaemia (updated)
Date of first decision: 14/12/2015, Withdrawn, Last updated: 01/12/2020 -
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Orphan designation: Recombinant adeno-associated viral vector serotype 5 encoding Staphylococcus aureus Cas9 endonuclease and two guide RNAs complementary to two regions of intron 26 of the CEP290 gene for: Treatment of Leber's congenital amaurosis (updated)
Date of first decision: 16/10/2017, Positive, Last updated: 30/11/2020 -
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Orphan designation: Recombinant human bone morphogenetic protein 4 for: Treatment of glioma (updated)
Date of first decision: 15/10/2014, Positive, Last updated: 30/11/2020 -
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Orphan designation: Obiltoxaximab for: Treatment of anthrax (updated)
Date of first decision: 24/08/2018, Positive, Last updated: 27/11/2020 -
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Orphan designation: Picropodophyllin for: Treatment of glioma (updated)
Date of first decision: 23/08/2017, Positive, Last updated: 27/11/2020 -
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Orphan designation: Synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues (lumasiran) for: Treatment of primary hyperoxaluria (updated)
Date of first decision: 21/03/2016, Positive, Last updated: 25/11/2020 -
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Orphan designation: Hydroxychloroquine for: Treatment of antiphospholipid syndrome (updated)
Date of first decision: 12/01/2017, Positive, Last updated: 24/11/2020 -
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Orphan designation: synthetic 15-amino-acid macrocyclic peptide acylated with a polyethyleneglycol palmitoylated linker (zilucoplan) for: Treatment of paroxysmal nocturnal haemoglobinuria (updated)
Date of first decision: 14/10/2016, Positive, Last updated: 24/11/2020 -
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Orphan designation: Humanised monoclonal antibody against P-selectin (crizanlizumab) for: Treatment of sickle cell disease (updated)
Date of first decision: 09/08/2012, Positive, Last updated: 24/11/2020 -
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Orphan designation: Acalabrutinib for: Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma (updated)
Date of first decision: 21/03/2016, Withdrawn, Last updated: 11/11/2020 -
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Orphan designation: Recombinant human beta-glucuronidase (vestronidase alfa) for: Treatment of mucopolysaccharidosis type VII (Sly syndrome) (updated)
Date of first decision: 21/03/2012, Positive, Last updated: 11/11/2020 -
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Orphan designation: Adeno-associated viral vector serotype rh10 containing the human factor IX gene for the treatment of haemophilia B- for: Treatment of haemophilia B (updated)
Date of first decision: 14/12/2015, Positive, Last updated: 11/11/2020 -
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Orphan designation: Triheptanoin for: Treatment of carnitine palmitoyltransferase II deficiency (updated)
Date of first decision: 28/07/2015, Positive, Last updated: 11/11/2020 -
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Orphan designation: Triheptanoin for: Treatment of mitochondrial trifunctional protein deficiency (updated)
Date of first decision: 28/07/2015, Positive, Last updated: 11/11/2020 -
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Orphan designation: Triheptanoin for: Treatment of long-chain 3-hydroxyacyl-coenzyme A dehydrogenase deficiency (updated)
Date of first decision: 28/07/2015, Positive, Last updated: 11/11/2020 -
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Orphan designation: Triheptanoin for: Treatment of very long-chain acyl-CoA dehydrogenase deficiency (updated)
Date of first decision: 19/06/2015, Positive, Last updated: 11/11/2020 -
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Orphan designation: adeno-associated viral vector serotype 8 containing the human glucose-6-phosphatase gene (pariglasgene brecaparvovec) for: treatment of glycogen storage disease type Ia (updated)
Date of first decision: 18/11/2016, Positive, Last updated: 11/11/2020 -
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Orphan designation: Adeno-associated viral vector serotype 8 encoding human ornithine transcarbamylase (avalotcagene ontaparvovec) for: Treatment of ornithine transcarbamylase deficiency (updated)
Date of first decision: 21/03/2016, Positive, Last updated: 11/11/2020 -
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Orphan designation: Setmelanotide for: Treatment of Prader-Willi syndrome (updated)
Date of first decision: 27/06/2016, Positive, Last updated: 09/11/2020 -
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Orphan designation: Setmelanotide for: Treatment of pro-opiomelanocortin deficiency (updated)
Date of first decision: 14/07/2016, Positive, Last updated: 09/11/2020 -
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Orphan designation: Setmelanotide for: Treatment of leptin receptor deficiency (updated)
Date of first decision: 19/11/2018, Positive, Last updated: 09/11/2020 -
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Orphan designation: Setmelanotide for: Treatment of Alström syndrome (updated)
Date of first decision: 09/01/2020, Positive, Last updated: 09/11/2020 -
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Orphan designation: Setmelanotide for: Treatment of Bardet-Biedl syndrome (updated)
Date of first decision: 21/08/2019, Positive, Last updated: 09/11/2020 -
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Orphan designation: humanised fusion protein consisting of extracellular domain of CD24 linked to IgG1 Fc domain for: Prevention of graft-versus-host disease (updated)
Date of first decision: 11/11/2015, Positive, Last updated: 09/11/2020 -
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Orphan designation: (3S)-3-{4-[7-(aminocarbonyl)-2H-indazol-2-yl] phenyl} piperidine tosylate monohydrate salt (niraparib) for: Treatment of ovarian cancer (updated)
Date of first decision: 04/08/2010, Positive, Last updated: 06/11/2020