PARIS (dpa-AFX) - Sanofi SA (SNY) said on Tuesday that its Phase 3 Baby-COMET study of Nexviazyme met its primary endpoint in treatment-naïve pediatric patients aged six months and younger with infantile-onset Pompe disease (IOPD).
The study showed that the proportion of participants who were alive and free of invasive ventilation after 52 weeks of treatment met the trial's main objective.
In addition, the trial achieved all secondary endpoints, including the proportion of participants alive and free of invasive ventilation at 12 and 18 months of age. Patients also demonstrated numerical improvements in other measures of disease progression after 52 weeks of treatment.
Nexviazyme was generally well tolerated in the study, with a safety profile consistent with previous experience. There were no serious treatment-related treatment-emergent adverse events, deaths, or treatment discontinuations. Infusion-associated reactions were reported in 29.4% of participants and were considered manageable.
Pompe disease is a rare, inherited, progressive neuromuscular disorder caused by the buildup of glycogen in the body's cells.
Nexviazyme is approved in several countries for the treatment of Pompe disease. In the United States, the therapy received approval in 2021 for 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 the long-term enzyme replacement therapy of both late-onset and infantile-onset Pompe disease.
Sanofi said it plans to use the Baby-COMET data to support a regulatory submission in the United States seeking approval of Nexviazyme for the treatment of infantile-onset Pompe disease.
Sanofi shares closed at $43.43 on Monday, up 1.12%.
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