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
August 31, 2026

Telomere Theraperutics SL

Orphan Drug Designation

Madrid
1-50 employees

treatment of idiopathic pulmonary fibrosis

Disease Area: Rare Diseases
August 30, 2026

AstraZeneca Pharmaceuticals LP

Orphan Drug Designation

treatment of esophageal cancer

Disease Area: Rare Diseases
August 27, 2026

Biomed Valley Discoveries Inc.

Orphan Drug Designation

Kansas City
1-50 employees

treatment of malignant glioma

Disease Area: Rare Diseases
August 27, 2026

Kalevala Therapeutics, Inc.

Orphan Drug Designation

Dover
1-50 employees

treatment of mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS)

Disease Area: Rare Diseases
August 27, 2026

Mirkine Pharma, Incorporated

Orphan Drug Designation

New York
1-50 employees

treatment of pancreatic cancer

Disease Area: Rare Diseases
August 26, 2026

RayzeBio, Inc.

Orphan Drug Designation

San Diego
1-50 employees

treatment of gastroenteropancreatic neuroendocrine tumors

Disease Area: Rare Diseases
August 26, 2026

X4 Pharmaceuticals, Inc.

Orphan Drug Designation

Boston
1-50 employees

treatment of congenital neutropenia

Disease Area: Rare Diseases
August 25, 2026

Kedrion S.p.A

Orphan Drug Designation

treatment of stiff person syndrome

Disease Area: Rare Diseases
August 24, 2026

SillaJen Inc.

Orphan Drug Designation

Seoul
1-50 employees

treatment of acute myeloid leukemia

Disease Area: Rare Diseases
August 24, 2026

LILA Holding AG

Orphan Drug Designation

Kusnacht
1-50 employees

treatment of Gaucher disease

Disease Area: Rare Diseases
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