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Orphan designations
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Last updated 17 days ago
Orphan designation: daratumumab Treatment of AL amyloidosis, 25/05/2018 Withdrawn
17 days ago
Orphan designation: daratumumab Treatment of AL amyloidosis, 25/05/2018 Withdrawn
Orphan designation: dasatinib Treatment of Diamond-Blackfan anaemia, 22/06/2026 Positive
19 days ago
Orphan designation: dasatinib Treatment of Diamond-Blackfan anaemia, 22/06/2026 Positive
Orphan designation: short hairpin RNA against NANOG mRNA Treatment of glioma, 23/06/2026 Positive
19 days ago
Orphan designation: short hairpin RNA against NANOG mRNA Treatment of glioma, 23/06/2026...
Orphan designation: satralizumab Treatment of myelin oligodendrocyte glycoprotein antibody-associated disease, 22/06/2026 Positive
19 days ago
Orphan designation: satralizumab Treatment of myelin oligodendrocyte glycoprotein antibody-associated disease, 22/06/2026 Positive
Orphan designation: humanised IgG4 monoclonal antibody against activated protein C Treatment of progressive multifocal leukoencephalopathy, 22/06/2026 Positive
19 days ago
Orphan designation: humanised IgG4 monoclonal antibody against activated protein C Treatment of...
Orphan designation: (S)-Quinuclidin-3-yl((R)-5-(3-chloro-4-isopropoxyphenyl)-2,2-dimethyl-2,3-dihydro-1H-inden-1-yl)carbamate ((1S,4R)-7,7-dimethyl-2-oxobicyclo[2.2.1]heptan-1-yl)methanesulfonate Treatment of Gaucher disease, 22/06/2026 Positive
19 days ago
Orphan designation: (S)-Quinuclidin-3-yl((R)-5-(3-chloro-4-isopropoxyphenyl)-2,2-dimethyl-2,3-dihydro-1H-inden-1-yl)carbamate ((1S,4R)-7,7-dimethyl-2-oxobicyclo[2.2.1]heptan-1-yl)methanesulfonate Treatment of Gaucher disease, 22/06/2026 Positive
Orphan designation: human IgG1 monoclonal antibody against JC polyomavirus, major capsid protein VP1 Treatment of progressive multifocal leukoencephalopathy, 22/06/2026 Positive
19 days ago
Orphan designation: human IgG1 monoclonal antibody against JC polyomavirus, major capsid protein...
Orphan designation: 3-(Aminocarbonyl)-1-(2,3,5-tri-O-acetyl-beta-D-ribofuranosyl)-pyridinium chloride (1:1) Treatment of ataxia telangiectasia, 22/06/2026 Positive
19 days ago
Orphan designation: 3-(Aminocarbonyl)-1-(2,3,5-tri-O-acetyl-beta-D-ribofuranosyl)-pyridinium chloride (1:1) Treatment of ataxia telangiectasia, 22/06/2026 Positive
Orphan designation: dinutuximab beta Treatment of Ewing sarcoma, 23/06/2026 Positive
19 days ago
Orphan designation: dinutuximab beta Treatment of Ewing sarcoma, 23/06/2026 Positive
Orphan designation: cyclo(arginyl-glycyl-aspartyl-D-tyrosyl-lysyl)-nizaracianine-1 Treatment of head and neck cancer, 22/06/2026 Positive
19 days ago
Orphan designation: cyclo(arginyl-glycyl-aspartyl-D-tyrosyl-lysyl)-nizaracianine-1 Treatment of head and neck cancer, 22/06/2026 Positive
Orphan designation: (S)-N-(2-Methoxy-5-((5-(trifluoromethyl)pyridin-2-yl)oxy)phenyl)-3-methyl-2-oxoimidazolidine-4-carboxamide Treatment of malignant mesothelioma, 22/06/2026 Positive
20 days ago
Orphan designation: (S)-N-(2-Methoxy-5-((5-(trifluoromethyl)pyridin-2-yl)oxy)phenyl)-3-methyl-2-oxoimidazolidine-4-carboxamide Treatment of malignant mesothelioma, 22/06/2026 Positive
Orphan designation: adeno-associated viral vector serotype 8 containing the human CASQ2 gene Treatment of catecholaminergic polymorphic ventricular tachycardia, 22/06/2026 Positive
20 days ago
Orphan designation: adeno-associated viral vector serotype 8 containing the human CASQ2 gene...