Pompe Disease Therapy Nexviazyme Clears Infant Phase 3, FDA Filing Set

Nexviazyme infantile-onset Pompe disease therapy cleared its Baby-COMET Phase 3 trial in newborns six months and younger, hitting every endpoint with no serious adverse events. Sanofi will file a U.S. label extension in the second half of 2026, targeting approval of avalglucosidase alfa as the first enzyme replacement therapy Phase 3-validated in this age group.