Sanofi: Nexviazyme Achieves Its Goal in Phase 3 for Infants
Sanofi announced on Tuesday positive phase 3 results for Nexviazyme in treating the early-infantile form of Pompe disease in infants. The company plans to submit these data to U.S. regulatory authorities by the second half of 2026.
Success Across All Expected Efficacy Criteria
The open-label Baby-COMET study, involving 17 treatment-naive infants aged 12 months or less, met its primary endpoint: the proportion of participants alive and free from invasive ventilation at 52 weeks. The trial also met all secondary endpoints, including maintaining this state of survival without respiratory assistance at 12 and 18 months of life, as well as measurable improvements in other disease progression indicators at 52 weeks. These clinical results will be presented on July 8, 2026, at the 19th International Congress of Neuromuscular Diseases, to be held in Florence. The tolerance profile was favorable: no serious adverse events related to the treatment, no deaths, no discontinuations due to toxicity, with infusion-related reactions observed in 29.4% of participants and deemed manageable.
Indication Extension and Regulatory Prospects
Nexviazyme, already approved in several countries for the late-onset form of Pompe disease, is thus targeting an indication extension to early-infantile forms. In the United States, the drug was authorized in 2021 for patients aged one year and older with the late-onset form. In Europe, marketed under the name Nexviadyme, it received approval in 2022 covering both late-onset and early-infantile forms. Sanofi plans to file a request for label expansion with the U.S. federal authorities in the second half of 2026.
