Sanofi's Nexviazyme Meets Goals in Phase 3 Study for Infants with Pompe Disease
Sanofi announced positive results from the Baby-COMET Phase 3 study, showing its drug Nexviazyme met all primary and secondary endpoints in infants with Pompe disease. The drug is being considered for regulatory submission in the US.
Sanofi announced on June 30, 2026, that its drug Nexviazyme (avalglucosidase alfa) met all primary and secondary endpoints in the Phase 3 Baby-COMET study for infants diagnosed with Pompe disease. The study's primary endpoint, survival and freedom from invasive ventilation at 52 weeks of treatment, was achieved in treatment-naïve infants aged zero to six months.
The study also demonstrated numerical improvements in other measures of disease progression at 52 weeks. All secondary endpoints, including survival and freedom from invasive ventilation at 12 and 18 months of age, were met. Sanofi plans to submit this data to support a regulatory application in the U.S. for the treatment of infantile-onset Pompe disease (IOPD) in the second half of 2026.
Pompe disease is a rare, inherited, progressive neuromuscular disorder caused by a deficiency in the acid alpha-glucosidase enzyme. IOPD represents the most aggressive form of the disease, potentially leading to severe and fatal complications without treatment. Nexviazyme is designed to facilitate enzyme uptake into cells, aiding in the clearance of excess glycogen that accumulates in muscle cells and can damage skeletal and cardiac muscles.
In the Baby-COMET study, Nexviazyme was found to be well-tolerated, with a safety profile consistent with the known profile of avalglucosidase alfa. No serious treatment-emergent adverse events, deaths, or discontinuations were reported. Infusion-associated reactions were manageable and occurred in 29.4% of participants.
The results are scheduled to be presented on July 8, 2026, at the 19th International Congress on Neuromuscular Diseases in Florence, Italy. Nexviazyme is already approved in multiple countries for Pompe disease treatment. In the U.S., it was approved in 2021 for late-onset Pompe disease, and in Europe (as Nexviadyme), it gained approval in 2022 for long-term enzyme replacement therapy for both late-onset and infantile-onset Pompe disease.