Sanofi Drug for Rare Neuromuscular Disease Hits Goals in Late-Stage Trial

By Adria Calatayud

Sanofi said its Nexviazyme drug met primary and secondary objectives in a late-stage clinical trial for the most aggressive variant of a rare neuromuscular condition called Pompe disease.

The French drugmaker said Tuesday that the study achieved its primary goal by showing Nexviazyme's potential to support ventilator-free survival after 52 weeks of treatment in babies of up to six months of age with Pompe disease during the first months of life.

Pompe disease is a rare genetic disease that has its most aggressive variant when it first appears in babies, resulting in severe and potentially fatal complications.

The trial also met all secondary objectives, including the proportion of participants alive and free of invasive ventilation at 12 and 18 months of age, as well as improvements in other metrics of disease progression at 52 weeks, Sanofi said.

The drug was well tolerated in the trial, the company added.

Sanofi said it intends to submit the data to seek U.S. approval for Nexviazyme as a treatment of infantile-onset Pompe disease.

In the U.S., the drug was approved in 2021 for the treatment for late-onset Pompe disease in patients one year of age and older, the company said.

Write to Adria Calatayud at adria.calatayud@wsj.com

(END) Dow Jones Newswires

June 30, 2026 01:44 ET (05:44 GMT)