The study tested the experimental medication, fordadistrogene movaparvovec, among about 100 boys aged 4 to 7 with the rare genetic condition. After one year of treatment, the research did not prove efficacy in the primary endpoint — motor function improvement — or in the secondary endpoints — 10-meter run/walk velocity and time to rise from floor velocity.
The safety profile was manageable, Pfizer said in a news release. The drugmaker’s phase 2 trial of fordadistrogene movaparvovec recently paused dosing after a child died.
In summer 2023, the FDA awarded Sarepta Therapeutics the first gene therapy for Duchenne muscular dystrophy.
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