A child has died in Pfizer’s phase 2 study of its gene therapy candidate for Duchenne muscular dystrophy, the rare genetic disorder that primarily affects boys, according to a patient advocacy group.
On 7 May, Parent Project Muscular Dystrophy published a letter it received from Pfizer announcing the patient’s death in the drugmaker’s Daylight study.
Becker’s Hospital Review reports that the phase 2 trial is researching the safety and efficacy of fordadistrogene movaparvovec among boys aged two to four. Test sites include hospitals in Florida, Utah, Pennsylvania and Australia.
While it investigates the death, Pfizer has paused dosing in its phase 3 Ciffreo study – which overlaps with Daylight, and tests the drug among boys aged four to eight. The company is continuing other trial activities.
“We do not yet have complete information and are working with the trial site investigator to understand what happened,” Pfizer said in the letter. “The patient received the investigational gene therapy, fordadistrogene movaparvovec, in early 2023.”
A Pfizer spokesperson said the death was reported as cardiac arrest, but the company has not verified the letter posted by Parent Project Muscular Dystrophy.
Duchenne muscular dystrophy is a progressive muscle degeneration, and symptom onset usually begins between the ages of two and three.
The median age of survival in males with DMD is 23.7 years.
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