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Tracking Rare Disease Drug Development Challenges Amid a Shifting Policy Environment
Rare diseases are defined as those which affect fewer than 200,000 people in the United States. Despite advancements in rare disease research and development due to incentives created in the Orphan Drug Act more than 40 years ago, many rare diseases still lack effective treatments due to the complex science and limited market incentives involved in their development. Of the 10,000+ known rare diseases, just 5% have an FDA-approved treatment. Incubate's Rare Disease Investment Tracker measures the impacts of a challenging policy environment on rare disease R&D and notes where passage of policies like the ORPHAN Cures Act have had a positive impact.
Delnaggar Biotech Group
Orphan Drug Designation
FDA orphan drug designation: treatment of high-grade anal intraepithelial neoplasia (HGAIN)
Nanjing RegeneCore Biotech Co.
Orphan Drug Designation
FDA orphan drug designation: treatment of acute myeloid leukemia
mAbTree Biologics AG
Orphan Drug Designation
FDA orphan drug designation: treatment of essential thrombocythemia
Pharmascience Inc.
Orphan Drug Designation
FDA orphan drug designation: treatment of pericarditis
Haichang Biotech Co.
Orphan Drug Designation
FDA orphan drug designation: treatment of osteosarcoma
Veloxis Pharmaceuticals
Orphan Drug Designation
FDA orphan drug designation: prevention of organ rejection in patients receiving a liver transplant
HemaCell Biotechnology Inc.
Orphan Drug Designation
FDA orphan drug designation: treatment of Bernard-Soulier syndrome
Novita Pharmaceuticals
Orphan Drug Designation
FDA orphan drug designation: treatment of pancreatic cancer
Academic Pharmaceuticals Inc.
Orphan Drug Designation
FDA orphan drug designation: treatment of Kawasaki disease
Novartis Pharmaceuticals Corporation
Orphan Drug Designation
FDA orphan drug designation: treatment of myasthenia gravis