According to a new announcement, fordadistrogene movaparvovec (previously known as PF-06939926), Pfizer’s investigational mini-dystrophin gene therapy, did not meet its primary end point of improvement in motor function among ambulatory patients with Duchenne muscular dystrophy (DMD) in the phase 3 CIFFREO study (NCT04281485).1 The company noted that it will continue to closely monitor all participants enrolled in CIFFREO, and will evaluate appropriate next steps for this program.
In the trial, the gene therapy failed to achieve the primary end point, which investigators assessed by the change from baseline in the North Star Ambulatory Assessment at 1 year following treatment. The findings also did not show a significant difference between patients treated with the therapy and placebo in the secondary end points, which included 10-meter run/walk velocity and time-to-rise from floor velocity. The company noted that the overall safety profile of the treatment was manageable, with mostly reports of mild to moderate adverse events (AEs), and that treatment-related serious AEs generally responded to clinical management.
“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,” Dan Levy, MD, PhD, development head for DMD at Pfizer, said in a statement.1 “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.”
