French healthcare company Sanofi S.A. (Euronext Paris:SAN) (Nasdaq:SNY) announced on Tuesday that its Phase 3 Baby-COMET study met its primary endpoint, with treatment-naïve infants aged six months and younger with infantile-onset Pompe disease alive and free of invasive ventilation after 52 weeks of Nexviazyme (avalglucosidase alfa) treatment.

The study also met all secondary endpoints, including survival without invasive ventilation at 12 and 18 months of age, while showing numerical improvements in additional measures of disease progression at 52 weeks.

Sanofi said Nexviazyme was well tolerated, with a safety profile consistent with previous findings, no serious treatment-related adverse events, deaths, or discontinuations, and manageable infusion-associated reactions reported in 29.4% of participants.

The company will present the results on 8 July 2026 at the 19th International Congress on Neuromuscular Diseases in Florence, Italy, and plans to use the data to support a US regulatory submission for an expanded indication in the second half of 2026.

Nexviazyme is approved in multiple countries for Pompe disease, but its use in infantile-onset Pompe disease remains under clinical investigation in the United States.