Sanofi has announced positive results from the Baby-COMET phase 3 study, which evaluated Nexviazyme (avalglucosidase alfa) in infants with infantile-onset Pompe disease (IOPD). The study met its primary endpoint and all secondary endpoints, and Sanofi intends to use the data to support a regulatory submission in the United States.
Nexviazyme is an enzyme replacement therapy (ERT) designed to improve uptake of the acid alpha-glucosidase (GAA) enzyme into cells and help clear the excess glycogen that damages muscle tissue. In Baby-COMET, the primary endpoint was the proportion of treatment-naïve infants six months of age and younger who were alive and free of invasive ventilation after 52 weeks of treatment. Secondary endpoints included survival free of invasive ventilation at 12 and 18 months of age, along with measures of cardiac and motor function.
Baby-COMET was a single-arm, open-label, international study in which 17 participants received Nexviazyme by intravenous infusion every other week. According to Sanofi, the therapy was well tolerated, with a safety profile consistent with previous experience and no serious treatment-related adverse events, deaths, or discontinuations. The results will be presented on July 8, 2026, at the 19th International Congress on Neuromuscular Diseases in Florence, Italy, and Sanofi anticipates filing a US regulatory submission for a label extension in the second half of 2026.
Nexviazyme is already approved in the United States for late-onset Pompe disease (LOPD) in patients one year of age and older, and in Europe—where it is known as Nexviadyme—for both LOPD and IOPD. Its use in IOPD is currently under clinical investigation in the US and has not yet been evaluated by the FDA.
Source
Press release: https://www.sanofi.com/en/media-room/press-releases/2026/2026-06-30-05-00-00-3319382
Clinical trial: https://clinicaltrials.gov/study/NCT04910776