SRPT - Pfizer Phase III DMD Gene Therapy Study Misses Goal | Benzinga
Pfizer (NYSE: PFE) announced that a phase III study on its mini-dystrophin gene therapy fordadistrogene movaparvovec for treating Duchenne muscular dystrophy ("DMD") failed to meet the primary endpoint as well as key secondary endpoints.
The global phase III study, CIFFREO is investigating fordadistrogene movaparvovec in patients 4-8 years of age with DMD. A progressive and degenerative disorder, DMD leads to weakness and wasting away of the body's muscles due to disruption of dystrophin production. The disease usually affects boys and is mostly diagnosed in early childhood.
The primary endpoint of the study was an improvement in motor function among the DMD boys treated with fordadistrogene movaparvovec compared to placebo. The primary endpoint was assessed by change in the North Star Ambulatory Assessment (NSAA) one year after treatment. NSAA is a rating scale used to measure motor abilities. The study also failed to show a significant difference between participants treated with fordadistrogene movaparvovec and placebo for the key secondary endpoints. The study's key secondary endpoints included 10-meter run/walk velocity and time to rise from floor velocity.
As regards the safety profile of the gene therapy, the adverse events were mostly mild to moderate in nature while the treatment-related serious adverse events responded to clinical management. Pfizer ...