Sanofi enzyme replacement therapy shows mixed results in Pompe study

Sanofi (NASDAQ:SNY) announces positive results from a Phase 3 clinical trial, COMET, evaluating avalglucosidase alfa, an enzyme replacement therapy, in patients with late-onset Pompe disease, an inherited disorder characterized by the buildup of glycogen in the body’s cells due to the dysfunction of a certain enzyme.

The study met the primary endpoint demonstrating non-inferiority (no worse than) to alglucosidase alfa (Lumizyme) as measured by the change in respiratory function but it failed to demonstrate superiority, the other primary objective.

Since superiority was not shown, statistical testing of the secondary endpoints was not done.

The safety profiles were comparable.

Development is ongoing.

https://seekingalpha.com/news/3583373-sanofi-enzyme-replacement-therapy-shows-mixed-results-in-pompe-study