Sanofi announced that its drug Nexviazyme met all primary and secondary goals in the Phase 3 Baby-COMET study. The trial treated infantile-onset Pompe disease (IOPD), the most aggressive form of the rare genetic disorder.
The drug achieved the primary goal of survival without invasive ventilation after 52 weeks. This outcome applied specifically to infants under six months of age. The study also reported a consistent and manageable safety profile.
Sanofi plans a U.S. regulatory submission based on these results in the second half of 2026. Pompe disease causes progressive muscle damage and often leads to fatal heart or breathing complications. Nexviazyme is already approved for late-onset Pompe disease in patients aged one year and older.