Pfizer Discontinues Muscle Disease Gene Therapy Trial Following Death

Pfizer Inc. halted a trial of an experimental gene therapy for muscular dystrophy after a child who received it died unexpectedly.

In an email, the company stated, “The patient, a little boy, had a cardiac arrest after receiving Pfizer’s one-time treatment last year”.

“The boy was part of a mid-stage study, now concluded, that enrolled children between the ages of 2 and 4”, the company continued.

The company went on to say, “Pfizer has paused administering the same gene therapy in a separate, final-stage study while it investigates the boy’s death”.

The study is looking into fordadistrogene movaparvovec, a gene therapy used to treat Duchenne muscular dystrophy, a rare and deadly muscle-wasting disease that primarily affects boys.

Pfizer stated, We are attempting to evaluate our investigational gene therapy while protecting the safety of the participants, which is our top priority”.

The final-stage trial included boys aged 4 and 8, and the goal was to see if the medication could reduce disease progression when compared to a placebo.

The study’s results are expected this year.

Sarepta Therapeutics Inc.’s competing gene therapy received fast approval in the United States last year.

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