May 7 – A young patient died of cardiac arrest after receiving Pfizer Inc’s experimental gene therapy for a muscle-wasting disease called Duchenne muscular dystrophy, the drugmaker told Reuters on Tuesday. tested in mid-term trials.
“A fatal serious adverse event, cardiac arrest, was reported by a participant in the Phase 2 DAYLIGHT study,” a company spokesperson told Reuters in an emailed response.
The trial is testing boys aged 2 to 3 years old with DMD, an inherited muscular dystrophy in which most patients lack the protein dystrophin that keeps muscles intact. The disease is estimated to affect 1 in 3,500 male births worldwide.
“The patient received the investigational gene therapy fordadistrogene movaparvovec in early 2023,” said a statement in a community letter from the drugmaker’s DMD gene therapy team and released by the nonprofit advocacy group.
Pfizer did not immediately respond to Reuters’ request to confirm the company’s community letter.
The study, which launches in August 2022 and is expected to be completed in early 2029, will follow all participants for five years after receiving the gene therapy, according to updated information from the company’s clinical trial registry.
The company said it was reviewing the data with an independent external data monitoring committee to understand potential causes.
The gene therapy candidate is also being tested in another late-stage DMD study called CIFFREO in boys ages 4 to 8 years old, according to a pipeline update on the drugmaker’s website.
This has no impact on our expectations for late-stage results, the company told Reuters in an email.
“We expect to begin preliminary analysis of the Phase 3 CIFFREO trial of fordadistrogene movaparvovec likely by the end of this month and share topline results soon,” it added.
This article was generated from automated news agency feeds without modifications to the text.