treatment of primary CoQ10 deficiency
All entries for: Orphan Drug Designation
February 15, 2026
New York University, on behalf of NYU Grossman School of Medicine
Orphan Drug Designation
Disease Area: Rare Disease
February 15, 2026
Elpiscience (Suzhou) Biopharma, Ltd.
Orphan Drug Designation
treatment of desmoid tumors
Disease Area: Rare Disease
February 14, 2026
InGel Therapeutics Inc.
Orphan Drug Designation
treatment of retinitis pigmentosa
Disease Area: Rare Disease
February 13, 2026
Zomagen Biosciences Ltd, a wholly owned subsidiary of Ventyx Biosciences, Ltd.
Orphan Drug Designation
treatment of pericarditis
Disease Area: Rare Disease
February 13, 2026
Bristol Myers Squibb
Orphan Drug Designation
Lawrenceville, NJ
10,001-50,000 employees
treatment of follicular lymphoma
Disease Area: Rare Disease
February 12, 2026
NorthSea Therapeutics B.V.
Orphan Drug Designation
treatment of intestinal failure associated liver disease
Disease Area: Rare Disease
February 12, 2026
FBD Biologics Limited
Orphan Drug Designation
treatment of gastric cancer
Disease Area: Rare Disease
February 11, 2026
MeiraGTx UK II Ltd
Orphan Drug Designation
treatment of inherited retinal dystrophy due to GUCY2D gene mutations
Disease Area: Rare Disease
February 11, 2026
Xadcera Biopharmaceutical (Suzhou) Co., Ltd.
Orphan Drug Designation
treatment of pancreatic cancer
Disease Area: Rare Disease
February 10, 2026
2A Biosciences, Inc.
Orphan Drug Designation
treatment of neurotrophic keratopathy
Disease Area: Rare Disease