Sanofi's Venglustat Gets FDA Priority Review

The United States — Food and Drug Administration has granted priority review to Sanofi's new drug application for venglustat, an investigational oral glucosylceramide synthase inhibitor, for the treatment of type 3 Gaucher disease (GD3). This rare lysosomal storage disorder currently lacks targeted therapies for its progressive neurological manifestations. If approved, venglustat would be the first treatment in the United States to address these neurological symptoms. The decision from the United States — Food and Drug Administration is anticipated by November 25, 2026. The application is supported by positive data from the Phase 3 LEAP2MONO study, which evaluated venglustat's efficacy and safety in adult and pediatric patients with neurological manifestations of GD3 whose systemic symptoms were stabilized with enzyme replacement therapy. Venglustat has also received breakthrough therapy, fast-track, and orphan designations from the United States — Food and Drug Administration, and orphan status in the European Union and Japan. Regulatory review is also underway in the European Union, with additional global filings planned by Sanofi in 2026.