Pfizer's gene therapy for Duchenne muscular dystrophy failed to improve motor function in a Phase 3 trial with ambulatory boys aged 4-7.
Pfizer's gene therapy for Duchenne muscular dystrophy, fordadistrogene movaparvovec, failed to meet its primary endpoint of improving motor function in a Phase 3 trial involving ambulatory boys aged 4 to 7 years. The therapy also did not show significant differences in key secondary goals compared to the placebo. Last month, Pfizer reported a young patient's death due to cardiac arrest after receiving the therapy in a mid-stage trial.
June 12, 2024
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