Sanofi's venglustat under priority review by the FDA for type 3 Gaucher disease

Sanofi announced that the U.S. Food and Drug Administration (FDA) has granted priority review to its new drug application for venglustat. This oral treatment targets the progressive neurological manifestations of Gaucher disease type 3 (MG3), a rare disorder.

If approved, venglustat would become the first treatment in the United States for this condition. The FDA decision is expected on November 25, 2026. Venglustat demonstrated promising efficacy in the phase 3 LEAP2MONO trial, with no new significant safety signals.

The investigational oral inhibitor has already received several FDA designations and is under review in Europe and Japan. Sanofi is continuing its global regulatory efforts to make venglustat available to patients with MG3.