Sarepta Therapeutics announced that the FDA has lifted its voluntary pause on shipments of ELEVIDYS (delandistrogene moxeparvovec) for ambulatory Duchenne muscular dystrophy patients, with the company set to resume shipping the gene therapy to healthcare sites immediately. This crucial decision came after the FDA conducted a swift and comprehensive review of available safety information, including an investigation into the death of an 8-year-old in Brazil, which was ultimately determined to be unrelated to ELEVIDYS treatment. Sarepta’s CEO, Doug Ingram, expressed immense satisfaction with the FDA's rapid review, emphasizing a shared commitment to the Duchenne community. While shipments for ambulatory patients are now cleared, Sarepta will continue to collaborate with the FDA to finalize the safety label update for ELEVIDYS and to establish a clear risk-mitigation approach for non-ambulatory patients, whose treatment remains on pause pending these ongoing discussions.

ELEVIDYS, positioned as the sole approved gene therapy for Duchenne, is indicated for individuals aged at least 4 years with a confirmed DMD gene mutation; however, continued approval for non-ambulatory patients hinges on the verification of clinical benefit in subsequent confirmatory trials. The company also highlighted important safety considerations for ELEVIDYS, including the potential for infusion-related reactions, acute serious liver injury, immune-mediated myositis, and myocarditis, along with contraindications for patients with specific DMD gene deletions and a recommendation against administration for those with high pre-existing anti-AAVrh74 antibody titers.