Sanofi Drug Shows Promise for Young Pompe Patients

Sanofi's investigational drug, Nexviazyme (avalglucosidase alfa), has shown significant promise for treating infantile-onset Pompe disease (IOPD) in very young children. The Phase III Baby-COMET trial successfully met its primary endpoint, demonstrating that treatment-naïve infants six months of age and younger with IOPD were alive and free of invasive ventilation after 52 weeks.

Further positive outcomes were observed across all secondary endpoints, indicating sustained ventilator-free survival at 12 and 18 months, along with improvements in disease progression metrics. Throughout the study, Nexviazyme exhibited good tolerability, with a safety profile aligning with its established use. No serious adverse events, deaths, or treatment discontinuations were noted, and infusion-related reactions were manageable.

Pompe disease is a rare, genetic disorder impairing muscle function due to a deficiency in the GAA enzyme. IOPD is its most severe form, requiring urgent intervention. The study's findings, to be presented on July 8, 2026, at the 19th International Congress on Neuromuscular Diseases in Florence, Italy, are expected to support a regulatory submission for label extension in the US during the latter half of 2026.

Nexviazyme is already approved in various countries for late-onset Pompe disease. This trial's success could significantly expand treatment options for the youngest and most vulnerable patients, offering hope where options are currently limited.