In June, Pifzer announced that their CIFFREO Phase 3 clinical trial for Duchenne muscular dystrophy (DMD) gene therapy did not meet its primary and secondary objectives. We now regret to inform you that the trial has been cancelled.
The CIFFREO Phase 3 clinical trial was a global study designed to test the safety and effectiveness of the gene therapy fordadistrogene movaparvovec in boys with DMD aged four to seven. This potential gene therapy aimed to deliver a version of the dystrophin gene, called mini dystrophin, into muscle cells. Individuals with DMD lack the dystrophin protein, which is crucial for maintaining muscle strength.
The results, detailed in Pfizer’s update on the study, showed that the trial failed to achieve its primary endpoint: demonstrating a significant improvement in motor function measured by a change in the NorthStar Ambulatory Assessment (NSAA) score. Additionally, the trial did not meet its secondary endpoints, which included the ten-metre run/walk velocity and time to rise from floor velocity.
Despite this disappointing outcome, the safety and well-being of all boys who received the gene therapy remain our top priority, and they will continue to be closely monitored.
Pfizer is currently reviewing the data collected from the trial and will share updates at future scientific meetings.
If you or a family member participated in the study, please contact your study site for further information.
