FDA Restricts Use of Sarepta's Gene Therapy, Adds Safety Warnings

Sarepta Therapeutics SRPT announced that the FDA has approved significant changes to the label of Elevidys, its one-shot gene therapy for Duchenne muscular dystrophy (DMD). The revised labeling not only narrows the eligible patient population but also adds new safety restrictions that further define how the therapy should be used.

Under the updated label, Elevidys is now approved only for ambulatory patients aged four years and older with DMD. It is no longer authorized for use in non-ambulatory patients. The gene therapy has been mandated to carry a boxed warning — the most important safety warning issued by the FDA — highlighting the risks of acute liver failure (ALF) and acute liver injury (ALI).

The agency also added additional limitations to Elevidys’ label. The gene therapy is no longer recommended for patients with pre-existing liver impairment, recent vaccinations or active/recent infections. The updated label also introduces new monitoring requirements, including weekly liver function tests for at least three months post-treatment. Patients are advised to remain near an appropriate medical facility for at least two months after infusion to ensure rapid access to care if complications arise.

These changes align with escalating safety concerns that have pressured Sarepta in recent months. Investor sentiment toward the stock has worsened significantly after two patient deaths were linked to Elevidys, both caused by ALF in non-ambulatory DMD patients. Before this label update, Sarepta had voluntarily suspended both clinical and commercial Elevidys dosing in this population.

As part of the postmarketing obligations, Sarepta is also required to conduct an observational study to further evaluate the risk of serious liver injury. The study will enroll roughly 200 DMD patients and follow them for at least 12 months after receiving Elevidys.

SRPT’s Stock Performance

Despite the substantial label changes, SRPT shares rose nearly 6% on Friday’s trading session. The positive move likely reflected relief among investors that Elevidys was not pulled from the market entirely, even with the added restrictions and boxed warning. The therapy remains Sarepta’s key commercial driver, and retaining market availability — even for a narrower patient population — appears to have eased some of the worst-case regulatory concerns.

Year to date, the stock has plummeted nearly 85% against the industry’s 15% growth.

More on Sarepta’s Elevidys

To address the safety issues observed in non-ambulatory patients, Sarepta has disclosed that it is developing a revised treatment protocol featuring an enhanced, sirolimus-based immunosuppressive regimen. The goal is to reduce the risk of acute liver complications and improve the safety profile of Elevidys in this higher-risk subgroup. The company plans to initiate a clinical study of this updated regimen soon, to eventually resume dosing in the non-ambulatory population pending FDA review.

Sarepta developed Elevidys in partnership with pharma giant Roche RHHBY. In 2019, the company and Roche entered into a licensing agreement to develop Elevidys. Per the agreement, Roche has exclusive rights to launch and market the therapy in non-U.S. markets.

SRPT’s Zacks Rank 

Sarepta currently carries a Zacks Rank #3 (Hold).

Sarepta Therapeutics, Inc. Price

 

Sarepta Therapeutics, Inc. price | Sarepta Therapeutics, Inc. Quote

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