赛诺菲的Nexviazeme在庞贝氏病研究中实现了主要目标

2026-06-30 13:21

Sanofi (SNY) said Tuesday that Nexviazyme met its primary endpoint in a phase 3 study of treatment-naïve infants six months of age and younger with infantile-onset Pompe disease, with participants alive and free of invasive ventilation at 52 weeks.
The study also met all secondary endpoints including ventilator-free survival metrics. Nexviazyme was well tolerated with no serious treatment-related adverse events or deaths, the company said.
Infusion-associated reactions occurred in 29.4% of participants, the drugmaker noted, as it now intends to submit the data to support a regulatory application in the U.S. for the treatment of infantile-onset Pompe disease.
Nexviazyme is approved in the US for late-onset Pompe disease patients one year and older.

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