On Tuesday, Sanofi SA (NASDAQ:SNY) reported data from the Phase 3 Baby-COMET study of Nexviazyme (avalglucosidase alfa).
Phase 3 Trial Hits Primary And Secondary Endpoints
The trial showed that Nexviazyme met its primary endpoint in treatment-naïve infants with infantile-onset Pompe disease (IOPD), supporting plans for a U.S. regulatory submission seeking a label expansion in the second half of 2026.
Pompe disease is a rare, inherited genetic disorder where glycogen builds up in the body’s cells. Caused by a deficiency of the enzyme acid alpha-glucosidase (GAA), it severely weakens skeletal and respiratory muscles.
The single-arm, open-label study evaluated infants aged six months or younger and found that the primary endpoint—the proportion of participants alive and free of invasive ventilation after 52 weeks of treatment—was achieved.
