A phase 3 trial of avalglucosidase alfa (Nexviazyme) in treatment-naïve infants with infantile-onset Pompe disease met its primary and all secondary endpoints. The Baby-COMET study showed improved ventilator-free survival, cardiac, and motor outcomes over 52 weeks with a favorable safety profile. Sanofi plans to submit for a US label expansion in the second half of 2026.
Sanofi's avalglucosidase alfa (Nexviazyme) has cleared every hurdle in the phase 3 Baby-COMET trial, meeting its primary and all prespecified secondary endpoints in treatment-naïve infants with infantile-onset Pompe disease (IOPD). The single-arm, open-label study enrolled 17 infants aged 12 months or younger, who received IV infusions every two weeks over 52 weeks. The results are set to be presented at the 2026 International Congress on Neuromuscular Diseases and will underpin a planned US regulatory submission for a label expansion — currently approved only for patients 1 year and older — expected in the second half of 2026.
IOPD is the most severe form of Pompe disease, a rare genetic lysosomal storage disorder. Without treatment, affected infants typically die within the first year of life from cardiorespiratory failure. Avalglucosidase alfa is engineered with approximately 15-fold greater mannose-6-phosphate content than the older standard-of-care enzyme, alglucosidase alfa, enabling more efficient glycogen clearance in target tissues.
Key Takeaways:
Why it matters: IOPD leaves families with very few options in the earliest, most critical months of life. These results could bring a more effective enzyme replacement therapy to the youngest and most vulnerable patients, potentially reshaping early intervention standards for this devastating disease.
