Sanofi's venglustat met primary and key secondary endpoints in the LEAP2MONO Phase 3 trial for type 3 Gaucher disease (GD3), showing neurological improvements and matching enzyme replacement therapy (ERT) on non-neurological outcomes like spleen volume, liver volume, and hemoglobin levels.123
Sanofi plans global regulatory submissions for venglustat in GD3 based on LEAP2MONO results.123
In the PERIDOT Phase 3 trial for Fabry disease, venglustat failed to meet the primary endpoint of reducing neuropathic and abdominal pain, though reductions were observed in both arms.123
An ongoing Phase 3 CARAT trial is evaluating venglustat's effect on left cardiac ventricular mass index in Fabry disease patients.123
Venglustat was well-tolerated in both trials with no new safety signals; common adverse events in LEAP2MONO included headache, nausea, spleen enlargement, and diarrhea.2
Sanofi markets existing treatments like Fabrazyme for Fabry and Cerezyme/Cerdelga for Gaucher disease.123
Sources:
1. https://www.biospace.com/drug-development/sanofi-rare-disease-drug-scores-a-hit-and-a-miss-in-phase-iii-tests
2. https://www.clinicaltrialsarena.com/news/sanofi-records-win-and-loss-with-rare-disease-drug-in-phase-iii-trials/
3. https://www.benzinga.com/markets/large-cap/26/02/50297319/sanofi-plans-regulatory-submission-for-investigational-drug-for-rare-disease-despite-mixed-data-from-pivotal-trials