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.

Date
Date
Disease Area
Announcement Type
March 30, 2026

Agiana Pharmaceuticals

Orphan Drug Designation

treatment of catecholaminergic polymorphic ventricular tachycardia

Disease Area: Rare Disease
March 26, 2026

Agnos Therapeutics

Orphan Drug Designation

treatment of retinitis pigmentosa

Disease Area: Rare Disease
March 26, 2026

Rege Nephro Co.

Orphan Drug Designation

treatment of autosomal dominant polycystic kidney disease

Disease Area: Rare Disease
March 25, 2026

Drug Farm

Orphan Drug Designation

treatment of retinal dystrophy, optic nerve edema, splenomegaly, anhidrosis, and migraine headache (ROSAH) syndrome

Disease Area: Rare Disease
March 24, 2026

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

March 24, 2026

ODDIFACT SAS

Orphan Drug Designation

treatment of myelin oligodendrocyte glycoprotein antibody-associated disease

Disease Area: Rare Disease
March 23, 2026

Anova Innovation Limited

Orphan Drug Designation

treatment of pancreatic cancer

Disease Area: Rare Disease
March 23, 2026

BlueRock Therapeutics LP

Orphan Drug Designation

treatment of cone-rod dystrophy

Disease Area: Rare Disease
March 23, 2026

USA

Orphan Drug Designation

treatment of hepatocellular carcinoma

Disease Area: Rare Disease
March 23, 2026

Shenzhen Hanhui Pharmaceutical Technology Co.

Orphan Drug Designation

treatment of idiopathic pulmonary fibrosis

Disease Area: Rare Disease
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