Pfizer Pauses Gene Therapy Trial For Muscle Disease After Death

Pfizer Inc. paused a study of an experimental gene therapy for muscular dystrophy after a child who received it died suddenly.

The patient, a young boy, suffered a cardiac arrest after receiving Pfizer's one-time treatment last year, the company said in an email. The boy was part of a mid-stage study, now concluded, that enrolled children between the ages of 2 and 4, the company said. 

Pfizer has paused administering the same gene therapy in a separate, final-stage study while it investigates the boy's death, the company said. That study is looking at the gene therapy, called fordadistrogene movaparvovec, to treat Duchenne muscular dystrophy, a rare and fatal muscle-wasting disease that primarily affects boys.

Pfizer said it's working to "evaluate our investigational gene therapy while protecting the safety of the participants, which is our top priority."

The final-stage trial enrolled boys between the ages of 4 and 8, testing whether the therapy could slow disease progression compared to a placebo. Results from that study are expected this year. 

A competing gene therapy from Sarepta Therapeutics Inc. won accelerated US approval last year.

The news was reported earlier by Stat, based on a letter posted on the website of the group Parent Project Muscular Dystrophy.

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