Sanofi's Nexviazyme meets all endpoints in infantile Pompe disease trial

Sanofi announced on June 30, 2026 that its enzyme therapy Nexviazyme (avalglucosidase alfa) met its primary endpoint in the Baby-COMET phase 3, single-arm, open-label study, measuring the proportion of treatment-naïve pediatric participants six months of age and younger with infantile-onset Pompe disease (IOPD) who were alive and free of invasive ventilation at 52 weeks of treatment.¹

The study also met all secondary endpoints, including the proportion of participants alive and free of invasive ventilation at 12 and 18 months of age, along with numerical improvements in other disease progression metrics at 52 weeks.¹

Sanofi said the data will support a regulatory submission for a label extension in the US, anticipated in the second half of 2026.¹ Results are set to be presented on July 8, 2026, at the 19th International Congress on Neuromuscular Diseases in Florence, Italy.¹

On safety, the company said Nexviazyme was well tolerated in the study, with safety consistent with the established profile of avalglucosidase alfa, no serious treatment-related treatment-emergent adverse events, deaths, or discontinuations, and manageable infusion-associated reactions in 29.4% of participants.¹

The trial design enrolled participants with IOPD 12 months of age and younger, with seventeen participants receiving intravenous Nexviazyme at 40 mg/kg every other week.¹ After a four-week screening period, participants received treatment for 52 weeks, followed by continued treatment for an additional 52 weeks and up to 104 additional weeks, with a four-week follow-up.¹

Nexviazyme currently carries US approval only for late-onset Pompe disease. In the US, Nexviazyme was approved in 2021 for the treatment of late-onset Pompe disease (LOPD) in patients one year of age and older.¹ Its use in IOPD remains under clinical investigation in the US, and its safety and efficacy in this indication have not been evaluated by the FDA.¹