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.”
BREAKING: A young boy died in a trial for Pfizer’s experimental gene therapy for Duchenne muscular dystrophy, the company told patient advocates Tuesday. – Stat News pic.twitter.com/qXeeRNApYQ
— Insider Paper (@TheInsiderPaper) May 7, 2024
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.
READ:
We are absolutely devastated to hear about the sudden passing of a young boy (2-3 years old) who was participating in Pfizer’s Phase 2 Daylight study! ⬇️
We are praying for peace and comfort for his family and friends. Fly high sweet Duchenne Warrior. pic.twitter.com/blZhLpqCoj
— “Sudden And Unexpected” (@toobaffled) May 7, 2024
Pfizer announced they are currently reviewing the patient’s data to understand what caused the boy to suffer an adverse event.
