Young participant in Pfizer's DMD gene therapy trial dies

A young boy participating in Pfizer's phase 2 gene therapy trial for Duchenne muscular dystrophy (DMD) has died. The boy was treated with Pfizer’s investigational gene therapy, fordadistrogene movaparvovec, as part of the Daylight trial. Following the incident, Pfizer has paused dosing in the crossover portion of another trial, CIFFREO, and is investigating the cause of the boy's death with regulatory and monitoring bodies.

The Daylight study, which continues despite the setback, aims to evaluate the safety and efficacy of the gene therapy, with results expected by the end of the year. This is not the first fatality linked to this treatment; another young male patient died in December 2021 during a phase 1 trial, leading to a temporary halt and FDA investigation. Pfizer remains committed to exploring the potential of fordadistrogene movaparvovec for DMD treatment.