Healthcare
Press Release: Sanofi’s Nexviazyme met all primary and secondary endpoints in infantile-onset Pompe disease phase 3 study
Sanofi’s Nexviazyme met all primary and secondary endpoints in infantile-onset Pompe disease phase 3 study Nexviazyme met its primary endpoint, participants alive and free of invasive ventilation, in treatment-naïve infants zero to six months of age in the Baby-COMET phase 3 study Sanofi intends to submit the data to support a regulatory application in the US for the treatment of infantile-onset Pompe disease Paris, June 30, 2026. Positive results from the Baby-COMET phase 3, single-arm, open-la
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Sanofi's Nexviazyme met all primary and secondary endpoints in infantile-onset Pompe disease phase 3 study Paris, June 30, 2026. Positive results from the Baby-COMET phase 3, single-arm, open-label study (clinical study identifier: NCT04910776), demonstrated that Nexviazyme (avalglucosidase alfa) met its primary endpoint: proportion of treatment-naïve pediatric participants 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. The results will be shared on July 8, 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. 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. In the Baby-COMET study, Nexviazyme was well tolerated and safety was consistent with the established profile of avalglucosidase alfa, with no serious treatment-related treatment-emergent adverse events, deaths, or discontinuations, and manageable infusion-associated reactions in 29.4% of participants. "Infantile-onset Pompe disease is a devastating, rapidly progressive condition that presents within the first days or weeks of life, making early intervention critical to help improve invasive ventilator-free survival beyond one year," said Priya S. Kishnani, MD, C.L. and Su Chen Professor of Pediatrics; Medical Director, ...