On Tuesday, Sanofi SA (NASDAQ:SNY) reported data from the Phase 3 Baby-COMET study of Nexviazyme (avalglucosidase alfa).
Phase 3 Trial Hits Primary And Secondary Endpoints
The trial showed that Nexviazyme met its primary endpoint in treatment-naïve infants with infantile-onset Pompe disease (IOPD), supporting plans for a U.S. regulatory submission seeking a label expansion in the second half of 2026.
Pompe disease is a rare, inherited genetic disorder where glycogen builds up in the body’s cells. Caused by a deficiency of the enzyme acid alpha-glucosidase (GAA), it severely weakens skeletal and respiratory muscles.
The single-arm, open-label study evaluated infants aged six months or younger and found that the primary endpoint—the proportion of participants alive and free of invasive ventilation after 52 weeks of treatment—was achieved.
The study also met all secondary endpoints, including the proportion of patients alive and free of invasive ventilation at 12 and 18 months of age. Researchers also reported numerical improvements across additional measures of disease progression after 52 weeks of treatment.
Safety Profile Remained Consistent
In the Baby-COMET study, Nexviazyme was generally well tolerated, with a safety profile consistent with the established experience for avalglucosidase alfa.
Researchers reported no serious treatment-related treatment-emergent adverse events, no deaths, and no treatment discontinuations. Infusion-associated reactions occurred in 29.4% of participants and were considered manageable.
Nexviazyme is approved in multiple countries for Pompe disease, although indications vary by region. The U.S. approved the drug in 2021for late-onset Pompe disease in patients aged one year and older.
In Europe, where it is marketed as Nexviadyme, it was approved in 2022 for long-term enzyme replacement therapy in both late-onset and infantile-onset Pompe disease.
SNY Stock Price Activity: Sanofi shares were down 1.32% at $42.85 at the time of publication on Tuesday, according to Benzinga Pro data.
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