FDA grants priority review to Sanofi's venglustat for type 3 Gaucher disease treatment

The FDA has accepted Sanofi's new drug application for venglustat, an oral treatment targeting neurological symptoms of type 3 Gaucher disease (GD3), for priority review with a decision expected by November 25, 2026. Venglustat is designed to cross the blood-brain barrier and address neuroinflammation caused by glycosphingolipid buildup in GD3 patients, a rare lysosomal storage disorder. The drug showed positive results in the phase 3 LEAP2MONO study, meeting primary and key secondary endpoints with a favorable safety profile. If approved, venglustat would be the first US treatment specifically for the neurological aspects of GD3, expanding Sanofi's rare disease portfolio and offering new hope for patients with this serious condition.