Pfizer’s Phase III DMD miss keeps Sarepta only game in town, for now

Pfizer’s DMD gene therapy misses primary and key secondary endpoints; data complicate surrogate endpoint questions

June 14, 2024 1:48 AM UTC

A Phase III miss for Pfizer’s Duchenne muscular dystrophy gene therapy is a blow to patients, and even with precedent for regulatory flexibility in the drug class, the path forward doesn’t look promising.

Pfizer Inc. (NYSE:PFE) reported that fordadistrogene movaparvovec missed the primary endpoint of change in the North Star Ambulatory Assessment (NSAA) at 52 weeks in a Phase III trial. It also missed key secondary endpoints including the 10-meter walk/run velocity and time to rise from floor velocity compared with placebo in the ambulatory Duchenne muscular dystrophy (DMD) patients 4-7 years old...