Pfizer Announces Phase 3 CIFFREO Trial Results for Gene Therapy in Duchenne Muscular Dystrophy

Pfizer today announced results from the Phase 3 CIFFREO trial evaluating fordadistrogene movaparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), in ambulatory boys aged 4-7 years. The trial did not meet its primary endpoint of improvement in the North Star Ambulatory Assessment (NSAA) score compared to placebo at 52 weeks. Despite these results, Pfizer observed increases in micro-dystrophin expression and some functional improvements in treated patients. The company will continue to analyze the data and work with regulatory authorities to determine next steps for this gene therapy program.