FX.co ★ Pfizer's Phase 3 Duchenne Gene Therapy Trial Fails To Meet Primary And Secondary Endpoints

Pfizer Inc. (PFE) announced that its Phase 3 global, multicenter, randomized, double-blind, placebo-controlled CIFFREO study, evaluating the investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec, in ambulatory patients with Duchenne muscular dystrophy (DMD), did not meet its primary endpoint. The study focused on boys aged 4 to 7 and aimed to assess improvement in motor function following treatment with the gene therapy compared to a placebo.

The primary endpoint was evaluated based on changes in the North Star Ambulatory Assessment one year post-treatment. Additionally, key secondary endpoints, such as 10-meter run/walk velocity and time to rise from the floor, failed to demonstrate a significant difference between those treated with fordadistrogene movaparvovec and the placebo group.

Despite these findings, Pfizer reported that the overall safety profile of fordadistrogene movaparvovec in the CIFFREO trial was manageable. Most adverse events were mild to moderate, and treatment-related serious adverse events generally responded well to clinical management.

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