Pfizer reports death of patient in Duchenne gene therapy trial

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A young boy died of cardiac arrest in a trial testing Pfizer’s experimental gene therapy for the muscle-wasting disease Duchenne muscular dystrophy (DMD). The drugmaker told on Tuesday.

“A fatal cardiac arrest was reported in a participant in the Phase 2 DAYLIGHT trial,” a company spokesman said in an email to .

The study tested boys ages 2 to under 4 with DMD, a genetic muscle-wasting disorder in which most patients lack the protein dystrophin, which keeps muscles intact. The disease is estimated to affect one in 3,500 male births worldwide.

“The patient received the gene therapy fordadistrogene movaparvovec in early 2023,” said a letter attributed to the drugmaker’s DMD gene therapy team and published by a nonprofit advocacy group.

The company said it is working with the independent third-party data monitoring board to review the data to understand the possible cause. (Reporting by Pratik Jain in Bengaluru; Editing by Shailesh Kuber)