Sanofi’s Nexviazyme (avalglucosidase alfa) could be set for approval in younger children with Pompe disease after the Phase III Baby-COMET trial met its primary endpoint.
In the single-arm, open-label study (NCT04910776), Nexviazyme met its primary endpoint of the proportion of treatment-naïve paediatric participants six months of age and younger with infantile-onset Pompe disease (IOPD) alive and free of invasive ventilation after 52 weeks of treatment.
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In addition, the study met all secondary endpoints, including the 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.
In the Baby-COMET study, Nexviazyme was well tolerated, and safety was consistent with its established profile, with no serious treatment-emergent adverse events (TEAEs), deaths, or discontinuations, and manageable infusion-associated reactions in 29.4% of pateints.
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.
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.
Dr Priya Kishnani, division chief, medical genetics of Duke University Medical Center, said: “IOPD 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. The Baby-COMET study shows the potential of Nexviazyme to support ventilator-free survival in infants, alongside encouraging cardiac and motor outcomes, offering important insights that may help advance the treatment landscape for these patients.”
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.
The drug is already 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.
There are very few drugs approved for use in Pompe Disease. In the US, there are just four drugs on the market, all of which are enzyme replacement therapies (ERTs) or combinations of enzyme therapies with stabiliser. This includes Nexviazyme.
