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 8, 2026

Mahzi Therapeutics

Orphan Drug Designation

treatment of Pitt Hopkins syndrome

Disease Area: Rare Disease
March 8, 2026

OcuNeo Therapeutics Inc.

Orphan Drug Designation

Sioux Falls
1-50 employees

treatment of vernal keratoconjunctivitis

Disease Area: Rare Disease
March 6, 2026

GENFIT

Orphan Drug Designation

treatment of acute-on-chronic liver failure

Disease Area: Rare Disease
March 6, 2026

Aminex Therapeutics, Inc.

Orphan Drug Designation

Kenmore
1-50 employees

prevention of allograft rejection in liver transplantation

Disease Area: Rare Disease
March 5, 2026

Aminex Therapeutics, Inc.

Orphan Drug Designation

Kenmore
1-50 employees

treatment of malignant glioma

Disease Area: Rare Disease
March 5, 2026

Multitude Therapeutics Inc.

Orphan Drug Designation

Shanghai
1-50 employees

treatment of malignant glioma

Disease Area: Rare Disease
March 5, 2026

Multitude Therapeutics Inc.

Orphan Drug Designation

Shanghai
1-50 employees

treatment of soft tissue sarcoma

Disease Area: Rare Disease
March 5, 2026

RNR Biomedical inc

Orphan Drug Designation

Tenafly
1-50 employees

treatment of Behcet disease

Disease Area: Rare Disease
March 2, 2026

Shanghai Allovanta Biotechnology Co., Ltd.

Orphan Drug Designation

Shanghai
1-50 employees

treatment of follicular lymphoma

Disease Area: Rare Disease
March 2, 2026

Takeda Development Center Americas, Inc.

Orphan Drug Designation

Cambridge
1-50 employees

treatment of narcolepsy type 1

Disease Area: Rare Disease
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