Pfizer Pauses Experimental Gene Therapy Trial After Boy’s ‘Sudden’ Death

Published on May 8, 2024

Pharmaceutical giant Pfizer Inc. has halted a study of an experimental gene therapy for muscular dystrophy after a child who received the treatment died due to cardiac arrest. The New York City-headquartered drugmaker revealed the boy’s sudden death in a community letter shared by the nonprofit advocacy group Parent Project Muscular Dystrophy (PPMD). The child—who was not identified by the company—received the investigational recombinant adeno-associated virus gene therapy known as fordadistrogene movaparvovec as part of Pfizer’s phase 2 “DAYLIGHT” study for Duchenne muscular dystrophy (DMD) in early 2023, the company said. According to Pfizer, the “DAYLIGHT” study enrolled children between the ages of two and four....