Sanofi Reports Positive Phase 3 Results for Nexviazyme in Infantile-Onset Pompe Disease

Phase 3 Study Meets Primary and Secondary Endpoints

Sanofi has announced positive topline results from its Baby-COMET Phase 3 clinical trial evaluating Nexviazyme (avalglucosidase alfa) in infants with infantile-onset Pompe disease (IOPD).

The study successfully met its primary endpoint by showing that treatment-naïve infants aged six months or younger remained alive and free from invasive ventilation after 52 weeks of treatment.

The trial also achieved all of its secondary endpoints. These included the proportion of patients alive and free from invasive ventilation at both 12 and 18 months of age, along with improvements in several other measures of disease progression after one year of treatment.

Results to Be Presented at International Medical Meeting

Sanofi plans to present the full Baby-COMET study findings on July 8, 2026, during the 19th International Congress on Neuromuscular Diseases in Florence, Italy.

The company also plans to use these results to support a regulatory submission for a label expansion in the United States. The submission is expected during the second half of 2026.

Understanding Infantile-Onset Pompe Disease

Pompe disease is a rare inherited genetic disorder caused by a deficiency of the acid alpha-glucosidase (GAA) enzyme. Without enough of this enzyme, glycogen builds up inside muscle cells, leading to progressive muscle damage throughout the body.

Infantile-onset Pompe disease is the most severe form of the condition. Symptoms usually appear within the first weeks or months of life and progress rapidly.

Without treatment, infants can develop serious complications affecting the heart, muscles, breathing, and movement. The disease can become life-threatening at an early age, making early diagnosis and treatment especially important.

How Nexviazyme Works

Nexviazyme is an enzyme replacement therapy designed to improve the delivery of the GAA enzyme into affected cells.

By increasing enzyme uptake, the treatment aims to remove excess glycogen from muscle cells, helping reduce damage to both skeletal muscles and the heart.

Researchers are studying whether this approach can improve survival and slow disease progression in infants with Pompe disease.

Safety Findings Remain Consistent

According to the Baby-COMET study, Nexviazyme demonstrated a safety profile consistent with previous clinical experience.

The trial reported no serious treatment-related adverse events, no treatment-related deaths, and no patient discontinued therapy because of side effects.

Some patients experienced infusion-associated reactions, affecting approximately 29.4% of participants. These events were reported to be manageable during treatment.

Experts Highlight the Importance of Early Treatment

Medical experts involved in the study noted that infantile-onset Pompe disease progresses very quickly, making early intervention critical.

They said the Baby-COMET results suggest that avalglucosidase alfa has the potential to improve ventilator-free survival while also showing encouraging effects on heart function and motor development in infants living with the disease.

The findings provide additional clinical evidence that could help improve treatment options for one of the most severe forms of Pompe disease.

Regulatory Status of Nexviazyme

Nexviazyme is already approved in several countries for the treatment of Pompe disease, although approved uses differ by region.

In the United States, the therapy received FDA approval in 2021 for the treatment of late-onset Pompe disease in patients aged one year and older.

In Europe, where the medicine is marketed as Nexviadyme, it was approved in 2022 for long-term enzyme replacement therapy in both late-onset and infantile-onset Pompe disease.

The use of Nexviazyme for infantile-onset Pompe disease in the United States remains under clinical investigation, and its safety and effectiveness for this indication have not yet been evaluated by the FDA.

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