Title: Pfizer’s Experimental Gene Therapy for Duchenne Muscular Dystrophy Linked to Patient Death
A young patient has tragically passed away due to cardiac arrest following the administration of Pfizer’s experimental gene therapy in a mid-stage trial for Duchenne muscular dystrophy (DMD), as reported by Reuters on May 7. The trial, known as the DAYLIGHT study, is testing the gene therapy on boys aged two to three with DMD, a genetic muscle wasting disorder that affects approximately one in 3,500 male births globally.
The patient received the investigational gene therapy, fordadistrogene movaparvovec, earlier this year. Pfizer has confirmed the incident, stating, “A fatal serious adverse event was reported as cardiac arrest for a participant in the Phase 2 DAYLIGHT study.” The company is currently reviewing the data with an independent external data monitoring committee to understand the potential cause.
Participants in the study will be followed for five years after treatment with gene therapy, which began in August 2022 and is estimated to complete in early 2029. Meanwhile, Pfizer’s gene therapy candidate is also being tested in another late-stage DMD study, called CIFFREO, involving boys aged 4 to less than 8 years old.
Despite the unfortunate event, Pfizer has stated that it does not expect this to impact the timeline for late-stage results. The company expects to begin the primary analysis of the Phase 3 CIFFREO trial of fordadistrogene movaparvovec at the end of May, with top-line results to be shared soon thereafter.
As the pharmaceutical industry continues to advance gene therapy research for DMD and other genetic disorders, incidents like these underscore the importance of rigorous safety measures and data review. Our thoughts are with the patient’s family during this difficult time.
