Sanofi has announced positive results from the Baby-COMET phase 3, single-arm, open-label study, showing that Nexviazyme (avalglucosidase alfa) met its primary endpoint, with a proportion of treatment-naïve infants of six months of age and younger with infantile-onset Pompe disease (IOPD) alive and free of invasive ventilation at 52 weeks of treatment.
In addition, the study met all secondary endpoints, including proportion of participants alive and free of invasive ventilation at 12 and 18 months of age, and numerical improvements in other metrics of disease progression at 52 weeks.
Pompe disease is a rare, inherited/genetic, progressive neuromuscular disease caused by a deficiency of the acid alpha-glucosidase (GAA) enzyme that affects muscle function throughout the body. IOPD constitutes the most aggressive variant of this disease, manifesting with swift symptom progression during the first months of life. Without therapeutic intervention, IOPD results in severe and potentially fatal complications affecting the heart, breathing and movement.
Nexviazyme is being evaluated as a potential treatment option for IOPD, designed to help enter cells and improve uptake of the essential GAA enzyme. This approach may help clear away excess glycogen, which builds up in muscle cells and can cause damage to skeletal and cardiac muscles.
Christopher Corsico, Global Head of Development at Sanofi, said: “These positive results offer the potential to expand access of Nexviazyme to more patients and families facing a condition with limited treatment options in the earliest months of life. The Baby-COMET findings are consistent with previous studies and reflect years of our scientific research aimed at translating deep biological understanding into clinical advances for the Pompe community.”
Nexviazyme is approved in multiple countries for the treatment of people living with Pompe disease, with specific indications varying by country. 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. In Europe, where the medicine is available under the name Nexviadyme, it received approval for the long-term enzyme replacement therapy of patients with Pompe disease (LOPD and IOPD) in 2022.
The results will be shared on 8 July 2026 at the 19th International Congress on Neuromuscular Diseases in Florence, Italy. In addition, the data will support a regulatory submission for a label extension in the US, anticipated in the second half of 2026.