News Updates
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.
Telomere Theraperutics SL
Orphan Drug Designation
Madrid
1-50 employees
treatment of idiopathic pulmonary fibrosis
AstraZeneca Pharmaceuticals LP
Orphan Drug Designation
treatment of esophageal cancer
Biomed Valley Discoveries Inc.
Orphan Drug Designation
Kansas City
1-50 employees
treatment of malignant glioma
Kalevala Therapeutics, Inc.
Orphan Drug Designation
Dover
1-50 employees
treatment of mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS)
Mirkine Pharma, Incorporated
Orphan Drug Designation
New York
1-50 employees
treatment of pancreatic cancer
RayzeBio, Inc.
Orphan Drug Designation
San Diego
1-50 employees
treatment of gastroenteropancreatic neuroendocrine tumors
X4 Pharmaceuticals, Inc.
Orphan Drug Designation
Boston
1-50 employees
treatment of congenital neutropenia
Kedrion S.p.A
Orphan Drug Designation
treatment of stiff person syndrome
SillaJen Inc.
Orphan Drug Designation
Seoul
1-50 employees
treatment of acute myeloid leukemia
LILA Holding AG
Orphan Drug Designation
Kusnacht
1-50 employees
treatment of Gaucher disease