News Updates
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. The Inflation Reduction Act's healthcare provisions are an example of policies that discourage investment in rare disease treatments, leading to discontinued research, layoffs, and ultimately delayed treatments for patients. Incubate's Rare Disease Investment Tracker measures the impacts of a challenging policy environment on rare disease R&D.
Calluna Pharma AS
Orphan Drug Designation
FDA orphan drug designation: treatment of idiopathic pulmonary fibrosis
Zenith Epigenetics Ltd.
Orphan Drug Designation
FDA orphan drug designation: treatment of nuclear protein in testis (NUT) carcinoma
Shanghai CirCode Biomed Co. Ltd.
Orphan Drug Designation
FDA orphan drug designation: treatment of thromboangiitis obliterans
Jazz Pharmaceuticals Ireland Limited
Orphan Drug Designation
FDA orphan drug designation: treatment of esophageal cancer
ArKri Therapeutics
Orphan Drug Designation
FDA orphan drug designation: treatment of Huntington’s disease
Curlim S.A.S
Orphan Drug Designation
FDA orphan drug designation: treatment of Charcot-Marie-Tooth disease (CMT)
Dewpoint Therapeutics Inc.
Orphan Drug Designation
FDA orphan drug designation: treatment of gastric cancer, including gastroesophageal junction cancer
Pangenix Pharma Limited
Orphan Drug Designation
FDA orphan drug designation: treatment of chronic pancreatitis
Schrödinger Inc.
Orphan Drug Designation
FDA orphan drug designation: treatment of Waldenstrom’s macroglobulinemia
Revolution Medicines Inc.
Orphan Drug Designation
FDA orphan drug designation: treatment of pancreatic cancer