FDA orphan drug designation: treatment of high-grade anal intraepithelial neoplasia (HGAIN)
All entries for: Rare Diseases
January 5, 2026
Nanjing RegeneCore Biotech Co.
Orphan Drug Designation
FDA orphan drug designation: treatment of acute myeloid leukemia
Disease Area: Rare Diseases
January 1, 2026
mAbTree Biologics AG
Orphan Drug Designation
FDA orphan drug designation: treatment of essential thrombocythemia
Disease Area: Rare Diseases
December 31, 2025
Pharmascience Inc.
Orphan Drug Designation
FDA orphan drug designation: treatment of pericarditis
Disease Area: Rare Diseases
December 31, 2025
Haichang Biotech Co.
Orphan Drug Designation
FDA orphan drug designation: treatment of osteosarcoma
Disease Area: Rare Diseases
December 23, 2025
Veloxis Pharmaceuticals
Orphan Drug Designation
FDA orphan drug designation: prevention of organ rejection in patients receiving a liver transplant
Disease Area: Rare Diseases
December 19, 2025
HemaCell Biotechnology Inc.
Orphan Drug Designation
FDA orphan drug designation: treatment of Bernard-Soulier syndrome
Disease Area: Rare Diseases
December 19, 2025
Novita Pharmaceuticals
Orphan Drug Designation
FDA orphan drug designation: treatment of pancreatic cancer
Disease Area: Rare Diseases
December 19, 2025
Academic Pharmaceuticals Inc.
Orphan Drug Designation
FDA orphan drug designation: treatment of Kawasaki disease
Disease Area: Rare Diseases
December 19, 2025
Novartis Pharmaceuticals Corporation
Orphan Drug Designation
FDA orphan drug designation: treatment of myasthenia gravis
Disease Area: Rare Diseases