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. 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.
CarnaBio USA, Inc.
Orphan Drug Designation
South San Francisco
1-50 employees
treatment of acute myeloid leukemia
Sumitomo Pharma America Inc.
Orphan Drug Designation
treatment of acute lymphoblastic leukemia
Cycle Pharmaceuticals Ltd.
Orphan Drug Designation
treatment of homocystinuria
Affinia Therapeutics
Orphan Drug Designation
treatment of dilated cardiomyopathy due to mutations in the BAG3 gene
Inhibikase Therapeutics, Inc.
Orphan Drug Designation
Wilmington, DE
1-50 employees
treatment of pulmonary arterial hypertension
Etnova Therapeutics Corp.
Orphan Drug Designation
treatment of hepatocellular carcinoma
Regeneron Pharmaceuticals Inc.
Orphan Drug Designation
treatment of Netherton syndrome
Nationwide Children’s Hospital
Orphan Drug Designation
treatment of neurofibromatosis type 2
Eye Care International LLC
Orphan Drug Designation
treatment of neurotrophic keratitis
Mimetogen Pharmaceuticals Inc.
Orphan Drug Designation
treatment of neurotrophic keratitis (NK)