Sanofi's Venglustat Meets Primary Endpoints in Phase 3 Study for Type 3 Gaucher Disease

Sanofi announced on February 2, 2026, that its investigational drug venglustat achieved all primary and three out of four key secondary endpoints in the Phase 3 LEAP2MONO study for type 3 Gaucher disease (GD3), a rare lysosomal storage disorder characterized by significant neurological symptoms.

Venglustat, an orally administered glucosylceramide synthase inhibitor, aims to target the neurological aspects of GD3 for which no approved treatments currently exist. The study demonstrated the drug's superiority over enzyme replacement therapy (ERT) in alleviating neurological symptoms, as measured by the SARA and RBANS assessment scales. The drug also showed comparable efficacy on non-neurological parameters, including changes in spleen volume, liver volume, and hemoglobin levels.

Sanofi plans to pursue global regulatory filings for venglustat for GD3. The company has a long-standing commitment to research and treatment for rare diseases. The study results are set to be presented at the 22nd annual WORLD Symposium.

Venglustat is also under investigation for Fabry disease, another rare lysosomal disorder. However, prior results from the Phase 3 PERIDOT study for Fabry disease did not show a clear superiority in patient-reported pain compared to ERT. A second Phase 3 study, CARAT, for Fabry disease treatment is still ongoing.

Venglustat's safety profile was generally well-tolerated. The most common adverse events in the venglustat arm included headache, nausea, spleen enlargement, and diarrhea, with some of these events being more frequent than in the comparator group. Sanofi will proceed with its development and prepare for regulatory submissions.