FDA Grants Priority Review for Sanofi’s Venglustat after Positive Phase III Results
SNY•FDA has granted priority review to Sanofi’s NDA for venglustat, targeting type 3 Gaucher disease, with a decision expected by November 25, 2026. The Phase III LEAP2MONO trial met both primary and three secondary endpoints, showing venglustat’s ability to cross the blood-brain barrier and address neurological symptoms.
1. FDA Priority Review Granted
Sanofi’s new drug application for venglustat, an oral glucosylceramide synthase inhibitor for type 3 Gaucher disease, has received FDA priority review under the six-month track, with a final decision due by November 25, 2026.
2. Phase III LEAP2MONO Trial Results
The double-blind, active-comparator LEAP2MONO trial enrolled 43 patients aged 12 and older, comparing venglustat plus placebo infusion against enzyme replacement therapy plus placebo tablet. The study met both modified SARA and RBANS primary endpoints and three of four systemic and biomarker secondary endpoints; common adverse events included headaches, nausea, spleen enlargement and diarrhoea.
3. Regulatory Status and Next Steps
Venglustat holds breakthrough therapy, fast-track and orphan designations in the US, and orphan status in the EU and Japan. The therapy is under review by EU regulators, and Sanofi plans further global filings this year to explore additional glycosphingolipid-related disorders beyond GD3.
