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
January 5, 2026

Delnaggar Biotech Group

Orphan Drug Designation

FDA orphan drug designation: treatment of high-grade anal intraepithelial neoplasia (HGAIN)

Disease Area: Rare Diseases
January 5, 2026

Nanjing RegeneCore Biotech Co.

Orphan Drug Designation

FDA orphan drug designation: treatment of acute myeloid leukemia

Disease Area: Rare Diseases
January 1, 2026

mAbTree Biologics AG

Orphan Drug Designation

FDA orphan drug designation: treatment of essential thrombocythemia

Disease Area: Rare Diseases
December 31, 2025

Pharmascience Inc.

Orphan Drug Designation

FDA orphan drug designation: treatment of pericarditis

Disease Area: Rare Diseases
December 31, 2025

Haichang Biotech Co.

Orphan Drug Designation

FDA orphan drug designation: treatment of osteosarcoma

Disease Area: Rare Diseases
December 23, 2025

Veloxis Pharmaceuticals

Orphan Drug Designation

FDA orphan drug designation: prevention of organ rejection in patients receiving a liver transplant

Disease Area: Rare Diseases
December 19, 2025

HemaCell Biotechnology Inc.

Orphan Drug Designation

FDA orphan drug designation: treatment of Bernard-Soulier syndrome

Disease Area: Rare Diseases
December 19, 2025

Novita Pharmaceuticals

Orphan Drug Designation

FDA orphan drug designation: treatment of pancreatic cancer

Disease Area: Rare Diseases
December 19, 2025

Academic Pharmaceuticals Inc.

Orphan Drug Designation

FDA orphan drug designation: treatment of Kawasaki disease

Disease Area: Rare Diseases
December 19, 2025

Novartis Pharmaceuticals Corporation

Orphan Drug Designation

FDA orphan drug designation: treatment of myasthenia gravis

Disease Area: Rare Diseases
Scroll to Top