Clinical TrialJul 7, 2026, 09:07 AM
Sanofi's Nexviazyme meets all endpoints in Pompe disease Phase 3 study
AI Summary
Sanofi announced positive results from its Baby-COMET Phase 3 study for Nexviazyme (avalglucosidase alfa) in infantile-onset Pompe disease (IOPD). The study met its primary endpoint, demonstrating that treatment-naïve infants (zero to six months of age) were alive and free of invasive ventilation at 52 weeks. All secondary endpoints were also met, and the drug was well tolerated with a safety profile consistent with previous findings. Sanofi plans to submit these data to support a regulatory application in the US for IOPD in the second half of 2026.
Key Highlights
- Nexviazyme met its primary endpoint in the Baby-COMET Phase 3 study for infantile-onset Pompe disease (IOPD).
- Primary endpoint: participants alive and free of invasive ventilation at 52 weeks in infants 0-6 months.
- The study also met all secondary endpoints, showing numerical improvements in disease progression metrics.
- Sanofi intends to submit data for a US regulatory application for IOPD in the second half of 2026.
- Nexviazyme was well tolerated with safety consistent with its established profile; no serious adverse events.
- Infusion-associated reactions occurred in 29.4% of participants, which were manageable.
- Nexviazyme is approved in the US for late-onset Pompe disease (LOPD) in patients one year and older.
- In Europe, Nexviadyme is approved for long-term enzyme replacement therapy for both LOPD and IOPD.
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