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Orphan designations
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Last updated 15 days ago
Orphan designation: diazoxide choline Treatment of Prader-Willi syndrome, 12/10/2017 Positive
15 days ago
Orphan designation: diazoxide choline Treatment of Prader-Willi syndrome, 12/10/2017 Positive
Orphan designation: aganirsen Prevention of corneal graft rejection, 20/04/2026 Positive
17 days ago
Orphan designation: aganirsen Prevention of corneal graft rejection, 20/04/2026 Positive
Orphan designation: surovatamig Treatment of large B-cell lymphoma, 20/04/2026 Positive
17 days ago
Orphan designation: surovatamig Treatment of large B-cell lymphoma, 20/04/2026 Positive
Orphan designation: 3-(4-((7-Cyano-1-methyl-2-((1-methyl-2-oxo-5-(trifluoromethyl)-1,2-dihydropyridin-3-yl)amino)-1H-imidazo[4,5-b]pyridin-6-yl)oxy)pyridin-2-yl)-1,1-dimethylurea Treatment of myelofibrosis, 20/04/2026 Positive
17 days ago
Orphan designation: 3-(4-((7-Cyano-1-methyl-2-((1-methyl-2-oxo-5-(trifluoromethyl)-1,2-dihydropyridin-3-yl)amino)-1H-imidazo[4,5-b]pyridin-6-yl)oxy)pyridin-2-yl)-1,1-dimethylurea Treatment of myelofibrosis, 20/04/2026 Positive
Orphan designation: adeno-associated virus serotype SLB101 containing the human microdystrophin gene Treatment of Duchenne muscular dystrophy, 20/04/2026 Positive
17 days ago
Orphan designation: adeno-associated virus serotype SLB101 containing the human microdystrophin gene Treatment...
Orphan designation: antisense oligonucleotide against SYNGAP1 regulatory RNA Treatment of SYNGAP1-related disorder, 20/04/2026 Positive
17 days ago
Orphan designation: antisense oligonucleotide against SYNGAP1 regulatory RNA Treatment of SYNGAP1-related disorder...
Orphan designation: upadacitinib Treatment of systemic sclerosis, 20/04/2026 Positive
17 days ago
Orphan designation: upadacitinib Treatment of systemic sclerosis, 20/04/2026 Positive
Orphan designation: coagulation factor V Treatment of congenital factor V deficiency, 20/04/2026 Positive
17 days ago
Orphan designation: coagulation factor V Treatment of congenital factor V deficiency, 20/04/2026...
Orphan designation: Momelotinib dihydrochloride monohydrate Treatment of VEXAS syndrome, 20/04/2026 Positive
17 days ago
Orphan designation: Momelotinib dihydrochloride monohydrate Treatment of VEXAS syndrome, 20/04/2026 Positive
Orphan designation: daraxonrasib Treatment of pancreatic cancer, 20/04/2026 Positive
17 days ago
Orphan designation: daraxonrasib Treatment of pancreatic cancer, 20/04/2026 Positive
Orphan designation: oremepermin alfa Treatment of spinal cord injury, 20/04/2026 Positive
17 days ago
Orphan designation: oremepermin alfa Treatment of spinal cord injury, 20/04/2026 Positive
Orphan designation: lisaftoclax Treatment of myelodysplastic syndromes, 13/04/2026 Positive
17 days ago
Orphan designation: lisaftoclax Treatment of myelodysplastic syndromes, 13/04/2026 Positive