Monday, May 27, 2024

Pfizer Halts Gene Therapy Trial Following Patient’s Death: Safety Concerns Arise

Pfizer Inc. has temporarily stopped a trial for an experimental gene therapy aimed at treating muscular dystrophy after the sudden death of a young patient who had received the treatment.

The patient, a young boy, suffered a cardiac arrest after receiving Pfizer’s one-time treatment last year, the company disclosed in an email. The trial, focused on testing the gene therapy called fordadistrogene movaparvovec, was part of a mid-stage study involving children between the ages of 2 and 4. While the mid-stage study has concluded, Pfizer has paused the same gene therapy’s administration in a separate, final-stage study, as it investigates the circumstances of the boy’s death. This final-stage trial, which enrolled boys between the ages of 4 and 8, aimed to determine whether the therapy could slow the progression of Duchenne muscular dystrophy, a rare and fatal muscle-wasting disease primarily affecting boys.

Pfizer emphasized that ensuring participant safety remains their top priority and that they are actively assessing their investigational gene therapy. The trial’s results, which were expected this year, are now pending while the investigation continues.

This decision comes following the accelerated US approval granted to a competing gene therapy developed by Sarepta Therapeutics Inc. last year. Reports on the trial’s suspension were initially disclosed by Stat, based on a letter posted on the website of the Parent Project Muscular Dystrophy group.

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