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

(RTTNews) - Pfizer Inc. (PFE) said that CIFFREO, a Phase 3 global, multicenter, randomized, double-blind, placebo-controlled study evaluating the investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec, in ambulatory patients with Duchenne muscular dystrophy (DMD) did not meet its primary endpoint of improvement in motor function among boys 4 to 7 years of age treated with the gene therapy compared to placebo.

The primary endpoint in the final analysis was assessed by change in the North Star Ambulatory Assessment at one year after treatment.

Key secondary endpoints, including 10-meter run/walk velocity and time to rise from floor velocity, also did not show a significant difference between participants treated with fordadistrogene movaparvovec and placebo.

The company noted that the overall safety profile of fordadistrogene movaparvovec in the CIFFREO trial was manageable, with mostly mild to moderate adverse events, and treatment-related serious adverse events generally responding to clinical management.

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