FDA orphan drug designation: treatment of malignant glioma
All entries for: Rare Disease
October 17, 2025
GSK
Orphan Drug Designation
London, UK
50,001+ employees
Disease Area: Rare Diseases
October 17, 2025
Leukogene Therapeutics Inc.
Orphan Drug Designation
FDA orphan drug designation: treatment of acute myelogenous leukemia
Disease Area: Rare Diseases
October 17, 2025
Cellenkos Inc
Orphan Drug Designation
FDA orphan drug designation: treatment of amyotrophic lateral sclerosis
Disease Area: Rare Diseases
October 17, 2025
ChainGen Biopharma Ltd.
Orphan Drug Designation
FDA orphan drug designation: treatment of myotonic dystrophy type 1 (DM1)
Disease Area: Rare Diseases
October 17, 2025
Halia Therapeutics Inc.
Orphan Drug Designation
FDA orphan drug designation: treatment of myelodysplastic syndromes
Disease Area: Rare Diseases
October 17, 2025
Quoin Pharmaceuticals Ltd.
Orphan Drug Designation
FDA orphan drug designation: treatment of Netherton syndrome
Disease Area: Rare Diseases
October 16, 2025
Zulia Biotech Inc.
Orphan Drug Designation
FDA orphan drug designation: treatment of Huntington’s disease
Disease Area: Rare Diseases
October 16, 2025
MavriX Bio LLC
Orphan Drug Designation
FDA orphan drug designation: treatment of Angelman syndrome
Disease Area: Rare Diseases
October 16, 2025
Kiniksa Pharmaceuticals GmbH c/o Kiniksa Pharmaceuticals Corp
Orphan Drug Designation
FDA orphan drug designation: treatment of pericarditis
Disease Area: Rare Diseases
October 16, 2025
Latus Bio
Orphan Drug Designation
FDA orphan drug designation: treatment of late infantile neuronal ceroid lipofuscinosis (CLN2) disease
Disease Area: Rare Diseases
