Pfizer has paused a study of an experimental gene therapy for muscular dystrophy following the death of a child who received the treatment. The pharmaceutical company revealed the boy’s sudden death in a letter shared by the nonprofit advocacy group Parent Project Muscular Dystrophy (PPMD).
The child, who received the gene therapy known as fordadistrogene movaparvovec as part of Pfizer’s phase 2 “DAYLIGHT” study for Duchenne muscular dystrophy (DMD), passed away due to cardiac arrest. The study enrolled children between the ages of two and four and has since concluded.
Pfizer also completed initial dosing for a placebo-controlled, randomized Phase 3 trial for its DMD therapy called CIFFREO in 2023. However, dosing associated with the cross-over portion of the CIFFREO trial has been paused following the boy’s death.
The company is working with regulators and the Data Monitoring Committee to investigate the cause of the boy’s death. Pfizer emphasized that the safety and well-being of patients in clinical trials remain a top priority and pledged to share more information with the medical and patient community.
While dosing in the CIFFREO trial has been paused, other trial activities are continuing as scheduled. Duchenne muscular dystrophy is a severe genetic condition characterized by progressive muscle degeneration and weakness caused by alterations to the dystrophin protein.
Symptoms of DMD typically manifest in early childhood and can lead to heart and respiratory muscle complications. Pfizer has faced previous incidents of death related to its gene therapy candidate, prompting regulatory actions. The company continues to collect data to evaluate the potential benefits of the therapy for DMD treatment.
News of the boy’s death comes after Pfizer’s CSO praised the gene therapy during an earnings call, highlighting promising results in clinical trials. The Epoch Times has reached out to Pfizer for further comment on the matter.
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