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
July 20, 2026

CarnaBio USA, Inc.

Orphan Drug Designation

South San Francisco
1-50 employees

treatment of acute myeloid leukemia

Disease Area: Rare Diseases
July 19, 2026

Sumitomo Pharma America Inc.

Orphan Drug Designation

treatment of acute lymphoblastic leukemia

Disease Area: Rare Diseases
July 17, 2026

Cycle Pharmaceuticals Ltd.

Orphan Drug Designation

treatment of homocystinuria

Disease Area: Rare Diseases
July 16, 2026

Affinia Therapeutics

Orphan Drug Designation

treatment of dilated cardiomyopathy due to mutations in the BAG3 gene

Disease Area: Rare Diseases
July 16, 2026

Inhibikase Therapeutics, Inc.

Orphan Drug Designation

Wilmington, DE
1-50 employees

treatment of pulmonary arterial hypertension

Disease Area: Rare Diseases
July 14, 2026

Etnova Therapeutics Corp.

Orphan Drug Designation

treatment of hepatocellular carcinoma

Disease Area: Rare Diseases
July 14, 2026

Regeneron Pharmaceuticals Inc.

Orphan Drug Designation

treatment of Netherton syndrome

Disease Area: Rare Diseases
July 14, 2026

Nationwide Children’s Hospital

Orphan Drug Designation

treatment of neurofibromatosis type 2

Disease Area: Rare Diseases
July 14, 2026

Eye Care International LLC

Orphan Drug Designation

treatment of neurotrophic keratitis

Disease Area: Rare Diseases
July 14, 2026

Mimetogen Pharmaceuticals Inc.

Orphan Drug Designation

treatment of neurotrophic keratitis (NK)

Disease Area: Rare Diseases
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