Pfizer shares update on DMD gene therapy clinical trial
Betsy Goodfellow | June 13, 2024 | News story | Research and Development | DMD, Musculo-skeletal disorder, Pfizer, duchenne muscular dystropy, gene therapy
Pfizer has announced an update from its phase 3 randomised, multicentre, double-blind, placebo-controlled CIFFREO trial, which assessed the investigational mini-dystrophin gene therapy, gordadistrogene movaparvovec, in ambulatory patients with Duchenne muscular dystrophy (DMD).
The company has shared that the trial did not meet its primary endpoint of improvement of motor function in boys aged four to seven years of age who were treated with the gene therapy, compared to placebo. This endpoint was measured by change in the North Star Ambulatory Assessment (NSAA) at one year after treatment.
Other key endpoints such as 10m run/walk velocity and time to rise from floor velocity, also showed no significant difference between those treated with fordadistrogene movaparvovec and placebo.
The drug’s safety profile was manageable, with mostly mild to moderate adverse events, and any treatment-related serious adverse events generally responding well to clinical management.
The company will continue to assess patients in the trial, while evaluating its next steps for this programme.
Dan Levy MD PhD, development head for Duchenne muscular dystrophy at Pfizer, commented: “We are extremely disappointed that these results did not demonstrate the relative improvement in motor function that we had hoped. We plan to share more detailed results from the study at upcoming medical and patient advocacy meetings, with the goal of ensuring that learnings from this trial can help improve future clinical research and development of treatment options that can improve care for boys living with Duchenne muscular dystrophy. We are grateful for the boys, their families, advocates, and the investigators who have participated in this research and the continuing effort to advance treatment options for this debilitating disease.”
Betsy Goodfellow
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