Sarepta Therapeutics will restart shipments of its gene therapy for some patients after U.S. regulators lifted a brief pause prompted by recent patient deaths.
The Food and Drug Administration (FDA) said the company can resume distribution of its drug Elevidys to young patients with Duchenne muscular dystrophy who are still able to walk. The pause had been requested after the deaths of two older teens who were receiving the therapy. However, the FDA clarified that the recent death of an 8-year-old boy was not linked to the drug.
Following the news, Sarepta’s shares surged more than 16% in after-hours trading, reaching $13.86. This marks the latest stock swing tied to the fate of its leading product.
Elevidys is the first FDA-approved gene therapy for Duchenne muscular dystrophy (DMD) — a rare, fatal disease that causes muscle loss in boys and young men. The therapy received accelerated approval in 2023 for a small group of young patients. The FDA later allowed expanded use in older boys, including those who had lost the ability to walk.
Regulators halted shipments after two teens died from acute liver injury, a known side effect. A third patient died while taking another Sarepta drug, leading to further safety reviews.
The FDA now says the company must provide additional safety data before resuming treatments for older, non-walking patients. “The FDA will continue to work with the sponsor regarding non-ambulatory patients, which remains subject to a voluntary hold,” the agency stated.
Analyst Andrew Tsai of Jefferies said the FDA’s decision improves short-term sales prospects for Elevidys. “The street will feel relieved about the situation, suggesting meaningful stock upside potential,” he told investors in a note.
Duchenne muscular dystrophy affects approximately one in every 3,500 to 5,000 male births worldwide. The disease progressively weakens muscles, often leaving patients in wheelchairs by their early teens and shortening life expectancy. Gene therapies like Elevidys are designed to target the underlying genetic cause, aiming to slow the progression of symptoms.
For now, only ambulatory patients — those still able to walk — can receive the treatment. Future decisions will depend on new safety research provided by Sarepta.
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