Sanofi announced on June 30, 2026, that Nexviazyme met all primary and secondary goals in its Phase 3 Baby-COMET study. The trial evaluated the drug in previously untreated infants under six months old with infantile-onset Pompe disease (IOPD). This condition is the most severe form of the rare genetic disorder.
The primary endpoint confirmed that all patients remained alive and free from invasive ventilation after 52 weeks of treatment. Nexviazyme was well-tolerated and demonstrated a consistent safety profile throughout the study.
Sanofi plans to submit the data for U.S. regulatory approval in the second half of 2026. The filing seeks to expand Nexviazyme's indication beyond its current use for late-onset Pompe disease.