Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today issued the following statement:
Just before 6:00 p.m. ET today, the U.S. Food and Drug Administration (FDA) issued a press release announcing an investigation into the death of an eight-year-old Duchenne muscular dystrophy (Duchenne) patient who had received ELEVIDYS (delandistrogene moxeparvovec) gene therapy.
The death of this patient was deemed unrelated to treatment with ELEVIDYS. As reported yesterday by Naomi Kresge at Bloomberg News:
Roche Holding AG says the recent death of a patient in Brazil who had been treated with gene therapy Elevidys for Duchenne muscular dystrophy is unrelated to the treatment.
* The boy wasn’t a clinical trial participant; reporting physician assessed his death as being unrelated to the gene therapy, Roche says in statement
* Death was reported to health authorities
* Roche, which markets Sarepta’s Duchenne treatment Elevidys outside the US, declines to comment on the boy’s age or details of the case
Sarepta reported this event to FDA on June 18, 2025, via the FDA’s postmarketing electronic database, FAERS.
At Sarepta, patient safety and well-being are always our top priority. We are committed to upholding the highest safety standards for all of our therapies, and do so in accordance with applicable law and commitment to full regulatory transparency.
ELEVIDYS is the only approved gene therapy for families and children devastated by Duchenne, a rare, progressive and ultimately fatal disease. We remain committed to working closely with the FDA to ensure that all decisions are grounded in science and the best interests of patients, considering the compelling need of these families to access disease-modifying therapy.
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