Sanofi's Venglustat Granted Priority Review for Type 3 Gaucher Disease

Sanofi's investigational drug, venglustat, has been accepted for priority review by the FDA for treating type 3 Gaucher disease (GD3). This disease is characterized by the accumulation of harmful molecules in various organs and the central nervous system, leading to severe neurological issues.

If approved, venglustat will be the first U.S. treatment targeting GD3’s neurological symptoms. The decision from the FDA is expected by November 25, 2026. The application is supported by the LEAP2MONO phase 3 study, which showed positive results in addressing both primary and secondary endpoints for GD3 patients.

Venglustat has previously received several regulatory designations and is undergoing review in the EU. Sanofi plans to expand global regulatory filings for venglustat later in 2026.