Pfizer Inc. (NYSE: PFE) has announced that a late-stage clinical trial for its investigational gene therapy, fordadistrogene movaparvovec, in children aged 4 to 7 with Duchenne muscular dystrophy (DMD) failed to meet its primary endpoint of improving motor function. Additionally, secondary endpoints related to motor function showed no significant improvement compared to the placebo group.
This marks the second major setback for the program, as it had already been placed on a dosing pause due to a fatal serious adverse event that occurred in a Phase II study involving children aged 2 to 3 years.
Despite these challenges, Pfizer has indicated that the safety profile of the gene therapy remained manageable throughout the trial.- Flcube.com
