“Pfizer’s Phase 3 Study on Duchenne Muscular Dystrophy Gene Therapy Falls Short of Primary Endpoint”
Pfizer Inc. (NYSE: PFE) has reported that its CIFFREO Phase 3 trial, which evaluated the investigational mini-dystrophin gene therapy, fordadistrogene movaparvovec, in ambulatory Duchenne muscular dystrophy (DMD) patients, did not achieve its primary goal of enhancing motor function in boys aged 4 to 7 compared to placebo. The assessment, based on the North Star Ambulatory Assessment (NSAA) at one year post-treatment, also showed no significant difference in key secondary endpoints like 10-meter run/walk velocity and time to rise from floor velocity between the treated and placebo groups.
The overall safety profile of fordadistrogene movaparvovec in the CIFFREO trial was manageable, with mostly mild to moderate adverse events, and serious adverse events related to treatment typically responded to clinical management.
Dr. Dan Levy, Pfizer’s Development Head for Duchenne muscular dystrophy, expressed disappointment in the results and emphasized the company’s commitment to sharing detailed findings from the study at upcoming medical and patient advocacy meetings. Pfizer aims to leverage insights gained from this trial to enhance future research and development efforts for DMD treatment. The company expressed gratitude to the participants, families, advocates, and investigators involved in advancing treatment options for this debilitating disease.
Pfizer will continue monitoring all study participants closely and is assessing the next steps for the program.
About the Fordadistrogene Movaparvovec Clinical Program:
CIFFREO is a Phase 3 global study evaluating the safety and efficacy of fordadistrogene movaparvovec gene therapy in ambulatory male participants aged 4 to 7 with a genetic diagnosis of DMD who are on a stable daily regimen of glucocorticoids. The trial’s primary endpoint is the change from baseline to one year in NSAA total score.
The CIFFREO study is currently paused for dosing following a fatal serious adverse event in the Phase 2 DAYLIGHT trial, which is assessing the therapy’s safety and tolerability in participants aged 2 to 3 with DMD.
About Duchenne Muscular Dystrophy:
DMD is a severe genetic disease characterized by progressive muscle degeneration and weakness, primarily affecting boys. Symptoms typically appear in early childhood and lead to loss of mobility and premature death. DMD is the most common form of muscular dystrophy worldwide.
About Pfizer:
Pfizer is committed to bringing therapies that significantly improve lives by applying science and global resources. The company collaborates with healthcare providers, governments, and communities to ensure access to quality healthcare worldwide. With 175 years of experience, Pfizer continues to innovate and make a difference for patients worldwide.
