USFDAJun 8, 2026, 06:11 AM
Sanofi's venglustat accepted for US priority review for Gaucher disease
AI Summary
Sanofi announced that the US Food and Drug Administration (FDA) has granted priority review to its new drug application (NDA) for venglustat, an investigational oral glucosylceramide synthase inhibitor, for the treatment of type 3 Gaucher disease (GD3). This marks a significant step as venglustat aims to be the first treatment in the US to address the progressive neurological manifestations associated with GD3. The FDA's target action date for a decision is November 25, 2026, following positive data from the Phase 3 LEAP2MONO study.
Key Highlights
- Sanofi's venglustat granted priority review by US FDA for type 3 Gaucher disease (GD3).
- Venglustat is an investigational oral glucosylceramide synthase inhibitor (GCSi).
- If approved, it would be the first US treatment for neurological manifestations of GD3.
- The target action date for the FDA decision is November 25, 2026.
- NDA supported by positive Phase 3 LEAP2MONO study data, meeting primary and key secondary endpoints.
- Venglustat previously received breakthrough therapy, fast-track, and orphan designations for GD3.
- The drug is designed to cross the blood-brain barrier to treat CNS accumulation of glycosphingolipids.
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