Sarepta’s Risky Gamble: Gene Therapy Stays on Market Despite FDA Request

When three patients die after taking a cutting-edge drug, most companies hit the brakes. Not Sarepta Therapeutics.

The Massachusetts-based biotech company is under fire for refusing a request from the U.S. Food and Drug Administration to stop selling its Duchenne muscular dystrophy gene therapy, Elevidys. The AP reported that the FDA made the request after three patients who received gene therapies from Sarepta died, all from liver complications linked to treatment.

Sarepta said no.

The company claims its internal review found no new safety concerns for younger patients with early-stage Duchenne’s disease, and it plans to keep distributing the drug to that group. Sarepta says it’s in ongoing talks with the FDA.

The FDA’s authority to pull a drug from the market exists, but the process can drag on for months or years. In most cases, a company will voluntarily comply with the agency’s request. That didn’t happen here.

FDA Commissioner Marty Makary issued a blunt statement saying the agency supports access to treatments for life-threatening conditions, but won’t hesitate to act when a clear safety risk emerges.

Elevidys first grabbed headlines in 2023 when it became the first gene therapy approved for Duchenne’s muscular dystrophy in the U.S. Even then, some FDA scientists openly questioned whether it actually worked. 

The drug, which delivers a one-time genetic fix, was initially greenlit for younger boys who could still walk. Its use later expanded to older patients and those in later stages of the disease.

But after two teenage boys died while taking Elevidys, Sarepta pulled back shipments for older patients. On Friday, the company confirmed a third death—a 51-year-old man in a separate clinical trial for a different muscular dystrophy treatment. Sarepta says that trial is now on hold.

All three deaths were linked to liver injury, a known risk listed in the drug’s official safety information.

Facing mounting criticism, Sarepta recently announced plans to add a bold warning label to Elevidys. The company also laid off a third of its staff, moves that only fueled skepticism about its priorities.

At the heart of the controversy is a question many patients and families are asking: Should a drug that might save lives also be allowed to risk them?

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