Sanofi: Venglustat Meets Primary Endpoints in Phase 3 for Gaucher's Disease
Sanofi announces that venglustat has met all primary endpoints in the LEAP2MONO phase 3 study for type 3 Gaucher's disease. The company will proceed with global regulatory filings for this experimental treatment.
Significant Improvements in Neurological Symptoms
In adult and pediatric patients (aged 12 and over) with type 3 Gaucher's disease, venglustat demonstrated statistically significant improvements in neurological symptoms measured by the SARA score and RBANS index at week 52 compared to enzyme replacement therapy. Venglustat also matched the performance of enzyme replacement therapy on non-neurological criteria, including changes in spleen volume, liver volume, and hemoglobin levels. Venglustat was generally well tolerated with no new safety signals compared to previous studies.
Global Regulatory Filings and Ongoing Studies
Sanofi will proceed with global regulatory filings for venglustat in type 3 Gaucher's disease. Venglustat is also being studied for the treatment of Fabry disease: the PERIDOT study did not meet its primary endpoint, while the CARAT study, assessing the effect on cardiac left ventricular mass index, is ongoing. Venglustat remains an experimental drug whose safety and efficacy have not been evaluated by a regulatory authority. Detailed results will be presented at the 22nd annual WORLDSymposium.
