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.
Agiana Pharmaceuticals
Orphan Drug Designation
treatment of catecholaminergic polymorphic ventricular tachycardia
Agnos Therapeutics
Orphan Drug Designation
treatment of retinitis pigmentosa
Rege Nephro Co.
Orphan Drug Designation
treatment of autosomal dominant polycystic kidney disease
Drug Farm
Orphan Drug Designation
treatment of retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and migraine headache (ROSAH) syndrome
EpiFrontier Therapeutics
Investment
Pennington NJ
1-50 employees
EpiFrontier Therapeutics received $32 million to advance EPF-001 for the treatment of sickle cell disease, a rare blood disorder.
City: Pennington
State: NJ
Investment Amount (In Millions): $32
ODDIFACT SAS
Orphan Drug Designation
treatment of myelin oligodendrocyte glycoprotein antibody-associated disease
Anova Innovation Limited
Orphan Drug Designation
treatment of pancreatic cancer
BlueRock Therapeutics LP
Orphan Drug Designation
treatment of cone-rod dystrophy
USA
Orphan Drug Designation
treatment of hepatocellular carcinoma
Shenzhen Hanhui Pharmaceutical Technology Co.
Orphan Drug Designation
treatment of idiopathic pulmonary fibrosis