Pfizer Inc. halted a trial of an experimental gene treatment for muscular dystrophy after a youngster who received it died unexpectedly.
The patient, a little boy, had a cardiac arrest after getting Pfizer’s one-time treatment last year, according to an email from the company. The boy was a participant in a now-concluded mid-stage trial that included children aged 2 to 4, according to the company.
Pfizer has halted the administration of the same gene therapy in a separate, final-stage study
Pfizer has halted administration of the same gene therapy in separate, final-stage research while it investigates the boy’s death, the firm announced. That study is looking into fordadistrogene movaparvovec, a gene therapy used to treat Duchenne muscular dystrophy, a rare and deadly muscle-wasting condition that mostly affects boys.
Pfizer stated that it is attempting to “evaluate our investigational gene therapy while protecting the safety of the participants, which is our top priority.”
The final-stage experiment included boys aged 4 to 8, and the goal was to see if the medication could reduce disease progression when compared to 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.
The news was reported earlier by Stat, based on a letter posted on the website of the group Parent Project Muscular Dystrophy.