treatment of acute myeloid leukemia
All entries for: Orphan Drug Designation
July 20, 2026
CarnaBio USA, Inc.
Orphan Drug Designation
South San Francisco
1-50 employees
Disease Area: Rare Diseases
July 19, 2026
Sumitomo Pharma America Inc.
Orphan Drug Designation
treatment of acute lymphoblastic leukemia
Disease Area: Rare Diseases
July 17, 2026
Cycle Pharmaceuticals Ltd.
Orphan Drug Designation
treatment of homocystinuria
Disease Area: Rare Diseases
July 16, 2026
Affinia Therapeutics
Orphan Drug Designation
treatment of dilated cardiomyopathy due to mutations in the BAG3 gene
Disease Area: Rare Diseases
July 16, 2026
Inhibikase Therapeutics, Inc.
Orphan Drug Designation
Wilmington, DE
1-50 employees
treatment of pulmonary arterial hypertension
Disease Area: Rare Diseases
July 14, 2026
Etnova Therapeutics Corp.
Orphan Drug Designation
treatment of hepatocellular carcinoma
Disease Area: Rare Diseases
July 14, 2026
Regeneron Pharmaceuticals, Inc.
Orphan Drug Designation
Tarrytown, NY
10,000+ employees
treatment of Netherton syndrome
Disease Area: Rare Diseases
July 14, 2026
Nationwide Children’s Hospital
Orphan Drug Designation
treatment of neurofibromatosis type 2
Disease Area: Rare Diseases
July 14, 2026
Eye Care International LLC
Orphan Drug Designation
treatment of neurotrophic keratitis
Disease Area: Rare Diseases
July 14, 2026
Mimetogen Pharmaceuticals Inc.
Orphan Drug Designation
treatment of neurotrophic keratitis (NK)
Disease Area: Rare Diseases