PARIS, June 30, 2026
Sanofi has announced that its enzyme replacement therapy Nexviazyme® (avalglucosidase alfa) successfully met all primary and secondary endpoints in the Baby-COMET Phase 3 clinical trial, marking a significant advancement in the treatment of infantile-onset Pompe disease (IOPD), one of the most severe and rapidly progressive inherited neuromuscular disorders. The positive findings position Nexviazyme as a promising therapeutic option for treatment-naïve infants aged six months or younger, demonstrating improved survival without invasive ventilation while also delivering encouraging cardiac and motor function outcomes. Based on these results, Sanofi plans to submit a regulatory application in the United States during the second half of 2026 seeking an expanded indication for Nexviazyme in infantile-onset Pompe disease. The study reinforces the company’s commitment to advancing therapies for rare genetic diseases through innovative enzyme replacement technologies and highlights the growing importance of early intervention in improving long-term outcomes for pediatric patients.
Phase 3 Baby-COMET Trial Meets All Clinical Endpoints
The Baby-COMET Phase 3, single-arm, open-label, international multicenter study evaluated Nexviazyme in 17 treatment-naïve infants aged 12 months or younger diagnosed with infantile-onset Pompe disease. The trial achieved its primary endpoint, demonstrating that participants remained alive and free of invasive ventilation after 52 weeks of treatment. In addition, all secondary endpoints were successfully met, including ventilator-free survival at both 12 and 18 months of age, along with measurable improvements in disease progression markers such as left ventricular mass Z-score, Alberta Infant Motor Scale scores, and urinary glucose tetrasaccharide levels.
Importantly, the therapy demonstrated a favorable safety profile, with no treatment-related serious adverse events, deaths, or treatment discontinuations, while infusion-associated reactions remained manageable throughout the study. These findings further strengthen the clinical evidence supporting Nexviazyme as a next-generation enzyme replacement therapy capable of improving outcomes for infants facing this devastating rare disease.
Early Intervention Offers Hope for Rare Genetic Disease
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Infantile-onset Pompe disease is a rare inherited disorder caused by a deficiency of the acid alpha-glucosidase (GAA) enzyme, leading to excessive glycogen accumulation within muscle cells and resulting in progressive damage to skeletal and cardiac muscles. Without treatment, affected infants frequently develop severe heart failure, respiratory complications, and significant motor impairment during their first year of life. Nexviazyme has been specifically engineered with enhanced mannose-6-phosphate receptor binding, enabling improved cellular uptake of the replacement enzyme and more effective glycogen clearance compared with earlier therapies.
According to investigators, the Baby-COMET findings highlight the importance of early diagnosis and immediate therapeutic intervention, offering the potential to significantly improve ventilator-free survival while preserving cardiac and motor function during the most critical period of disease progression. These encouraging clinical outcomes provide new hope for patients and families facing a condition with historically limited treatment options.
Regulatory Expansion Supports Sanofi’s Rare Disease Strategy
Following the successful completion of the Baby-COMET trial, Sanofi intends to submit the clinical data to the U.S. Food and Drug Administration in support of a label expansion for infantile-onset Pompe disease later this year. Nexviazyme is already approved in multiple countries for the treatment of Pompe disease, including late-onset Pompe disease in the United States and both late-onset and infantile-onset Pompe disease in Europe under the brand name Nexviadyme. The latest Phase 3 results further reinforce Sanofi’s strategy of expanding access to innovative therapies for rare diseases while strengthening its leadership in enzyme replacement technologies. As rare disease research continues to evolve, the successful Baby-COMET study represents another important milestone in advancing precision therapies capable of addressing serious unmet medical needs and improving survival and quality of life for pediatric patients worldwide.
Source: Sanofi press release
