Overview
- The oral therapy met the primary endpoint and three of four key secondary measures in type 3 Gaucher disease, showing superior neurological improvements versus enzyme replacement therapy.
- The Fabry disease study did not achieve a statistically significant benefit on patient‑reported pain compared with placebo, though reductions in plasma lyso‑GL‑3 were observed and analyses continue.
- Sanofi characterized the Gaucher study’s safety profile as well tolerated, with higher rates of nausea, diarrhea and spleen enlargement among those receiving venglustat.
- The company said it intends to submit the Gaucher results to global regulators to determine next steps toward a potential approval.
- Venglustat, inherited from Genzyme, has a history of trial failures in other conditions, and analysts currently maintain muted commercial expectations despite the Gaucher result.