Sarepta Therapeutics Suspends Gene Therapy Shipments After Second Death

Sarepta Therapeutics has announced a temporary suspension of shipments for its gene therapy product, Elevidys® (delandistrogene moxeparvovec-rokl), following the tragic death of a second patient with Duchenne muscular dystrophy (DMD) in a span of three months. This decision has raised significant concerns regarding the safety and efficacy of gene therapies in treating this debilitating condition, particularly for non-ambulatory patients.

The announcement, made on June 15, 2025, follows the death of a non-ambulatory patient who succumbed to acute liver failure (ALF) after receiving Elevidys treatment. This incident marks the second reported fatality linked to the therapy, with the first being a 16-year-old male patient who died in March 2025. Louise Rodino-Klapac, PhD, the Chief Scientific Officer of Sarepta, expressed profound sadness over the loss, emphasizing the company's commitment to patient safety.

"We are deeply saddened by the loss of a second patient and extend our heartfelt condolences to the patient’s family and his care team during this incredibly difficult time," Rodino-Klapac stated. The company has implemented a temporary pause on shipments of Elevidys for non-ambulatory patients as it evaluates a new immunosuppressive regimen designed to mitigate the risk of liver complications.

In a proactive measure, Sarepta plans to convene an independent panel of leading experts in Duchenne and liver health to assess the proposed regimen and its potential to reduce liver enzyme elevations. The proposed regimen includes sirolimus, which has shown promise in preclinical studies. Sarepta is committed to sharing the panel's recommendations with the U.S. Food and Drug Administration (FDA) and ensuring any new protocols are compliant with FDA regulations.

This decision to pause shipments comes amid ongoing scrutiny of Elevidys, which is the first gene therapy approved by the FDA for DMD treatment. Initially granted accelerated approval in 2023 for ambulatory boys aged 4 to 5, its label was expanded last year to include all DMD patients aged 4 and older. However, the continued approval of Elevidys for non-ambulatory patients hinges on verifying clinical benefits in ongoing trials, notably the Phase III ENVISION trial (SRP-9001-303).

The FDA's former director of the Center for Biologics Evaluation and Research, Dr. Peter Marks, faced controversy over the approval process of Elevidys, having overridden staff objections to grant the therapy accelerated approval. Dr. Marks' abrupt resignation in March 2025 has fueled further discourse regarding the safety and regulatory oversight of gene therapies.

Recent financial reports reveal that Elevidys generated $375 million in net product revenue during the first quarter of 2025, a significant increase from the previous year. However, analysts predict that ongoing safety concerns may dampen future revenue growth. Jefferies equity analyst Andrew Tsai noted that the recent deaths could lead doctors to hesitate in prescribing Elevidys, potentially impacting overall sales.

"Our doctors, who were previously unfazed by the first patient death, might now reconsider their stance," Tsai commented, indicating that families might opt to deny treatment altogether rather than delay it. The implications of these events extend beyond immediate patient care, as they may influence investor confidence in gene therapy ventures broadly.

As the company navigates these challenges, the focus remains on patient safety and the evaluation of new treatment protocols. The ongoing discussions with regulatory bodies and expert panels highlight the complexities of advancing gene therapies in a landscape marked by both hope and caution. The future of Elevidys and similar gene therapies will depend significantly on the outcomes of these evaluations and the willingness of the medical community to embrace new treatment avenues amid emerging safety concerns.