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.

Date
Date
Disease Area
Announcement Type
October 24, 2025

Calluna Pharma AS

Orphan Drug Designation

FDA orphan drug designation: treatment of idiopathic pulmonary fibrosis

Disease Area: Rare Diseases
October 24, 2025

Zenith Epigenetics Ltd.

Orphan Drug Designation

FDA orphan drug designation: treatment of nuclear protein in testis (NUT) carcinoma

Disease Area: Rare Diseases
October 23, 2025

Shanghai CirCode Biomed Co. Ltd.

Orphan Drug Designation

FDA orphan drug designation: treatment of thromboangiitis obliterans

Disease Area: Rare Diseases
October 23, 2025

Jazz Pharmaceuticals Ireland Limited

Orphan Drug Designation

FDA orphan drug designation: treatment of esophageal cancer

Disease Area: Rare Diseases
October 22, 2025

ArKri Therapeutics

Orphan Drug Designation

FDA orphan drug designation: treatment of Huntington’s disease

Disease Area: Rare Diseases
October 22, 2025

Curlim S.A.S

Orphan Drug Designation

FDA orphan drug designation: treatment of Charcot-Marie-Tooth disease (CMT)

Disease Area: Rare Diseases
October 22, 2025

Dewpoint Therapeutics Inc.

Orphan Drug Designation

FDA orphan drug designation: treatment of gastric cancer, including gastroesophageal junction cancer

Disease Area: Rare Diseases
October 22, 2025

Pangenix Pharma Limited

Orphan Drug Designation

FDA orphan drug designation: treatment of chronic pancreatitis

Disease Area: Rare Diseases
October 21, 2025

Schrödinger Inc.

Orphan Drug Designation

FDA orphan drug designation: treatment of Waldenstrom’s macroglobulinemia

Disease Area: Rare Diseases
October 21, 2025

Revolution Medicines Inc.

Orphan Drug Designation

FDA orphan drug designation: treatment of pancreatic cancer

Disease Area: Rare Diseases
Scroll to Top