Children's Hospital LA pauses use of Sarepta gene therapy for all patients
The FDA on Friday asked Sarepta to voluntarily halt shipments of the gene therapy after a muscular dystrophy patient who received a different, experimental treatment died. The company said it did not plan to comply with the request and would continue making the treatment available to ambulatory patients.
The company in June suspended use of Elevidys for patients whose disease had progressed to the point where they need a wheelchair after two teenagers died from liver failure after treatment.
Elevidys has full FDA approval for children with Duchenne Muscular Dystrophy who are still able to walk, but was given conditional approval for non-ambulatory patients.
Children's Hospital LA said its pause, effective July 18, has been communicated to "affected patient families while it awaits any further determination by the FDA."
The hospital is one of more than 70 authorized to administer Elevidys. Five other major children's hospitals asked by Reuters to comment on whether they planned to continue use of the gene therapy did not immediately respond.
Shares of Sarepta, down about 90% year-to-date, fell 5% on Monday to close at $13.32 on Nasdaq.
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