Pfizer Reports Young Boy Dies From Cardiac Arrest After Participating in Gene Therapy Study

Pharmaceutical giant Pfizer has reported a young boy has died due to cardiac arrest after receiving the company’s experimental gene therapy for Duchenne muscular dystrophy.

A Pfizer spokesperson told Reuters in an exclusive email, “A fatal serious adverse event was reported as cardiac arrest for a participant in the Phase 2 DAYLIGHT study.”

Pfizer’s gene therapy trial is currently testing children two to three-year-olds who have Duchenne muscular dystrophy.

According to Johns Hopkins Medicine, Duchenne muscular dystrophy (DMD) “is caused by a genetic problem in producing dystrophin, a protein that protects muscle fibers from breaking down when exposed to enzymes.”

Per Reuters:

A young patient died due to cardiac arrest after receiving Pfizer’s gene therapy being tested in a mid-stage trial for a muscle-wasting disorder called Duchenne muscular dystrophy(DMD), the drugmaker told Reuters on Tuesday.

“A fatal serious adverse event was reported as cardiac arrest for a participant in the Phase 2 DAYLIGHT study,” a company spokesperson told Reuters in an emailed response.

The trial is testing boys 2 to three years of age with DMD, a genetic muscle wasting disorder in which most patients lack the protein dystrophin which keeps muscles intact. The disorder affects an estimated one-in-3,500 male births worldwide.

“The patient received the investigational gene therapy, fordadistrogene movaparvovec, in early 2023,” as per a statement from a community letter attributed to the drugmaker’s DMD gene therapy team and posted by a nonprofit advocacy group.

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Pfizer announced they are currently reviewing the patient’s data to understand what caused the boy to suffer an adverse event.

 

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