Sarepta Therapeutics Stock Plummets Over 40% On Second Fatal Case Linked To Its Gene Therapy

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) stock experienced a steep decline of approximately 40% in premarket trading on Monday following a sobering safety update for its Duchenne muscular dystrophy (DMD) gene therapy, Elevidys.

What Happened

On Sunday, Sarepta provided a safety update regarding Elevidys (delandistrogene moxeparvovec-rokl), the only approved gene therapy for patients with Duchenne muscular dystrophy, and steps the company is taking to strengthen the safety profile in non-ambulatory patients.

These steps follow a second reported case of acute liver failure (ALF), resulting in death.

In March, Sarepta Therapeutics reported a patient death following treatment with Elevidys, having suffered acute liver failure.

The cases of ALF to date have both occurred in non-ambulatory (patients who cannot walk independently) individuals with Duchenne.

Sarepta and Roche Holdings AG (OTC:RHHBY) temporarily halted several clinical studies in April.

During its first-quarter earnings release, Sarepta revised its 2025 total net product revenues guidance to $2.3-$2.6 billion from $2.9-$3.1 billion.

William Blair writes that the revised guidance primarily concerns delayed Elevidys turnaround times.

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Key Safety Initiatives

As part of a comprehensive review of safety data, Sarepta is taking proactive steps to mitigate the risk of acute liver failure in non-ambulatory patients.

A panel will evaluate data and assess the company’s proposed regimen, which includes sirolimus and is supported by preclinical data demonstrating the effectiveness of additional immunosuppression in moderating liver enzyme elevations, a key factor in mitigating potential safety events.

Sarepta will share the panel’s recommendations with the U.S. Food & Drug Administration (FDA), and implementation of any new regimen will be subject to FDA guidance and allowance.

In the meantime, Sarepta is temporarily suspending shipments of Elevidys for non-ambulatory patients.

For ambulatory patients, no treatment changes are being proposed and the current practice of administering corticosteroids before and after Elevidys infusion and post-treatment monitoring remains the same.

Sarepta has voluntarily paused dosing in the ENVISION clinical study (also known as Study SRP-9001-303).

ENVISION is a global, randomized, double-blind, placebo-controlled trial evaluating Elevidys in older ambulatory and non-ambulatory Duchenne muscular dystrophy patients.

In the U.S., it serves as the confirmatory trial required under the FDA’s accelerated approval pathway for non-ambulatory patients.

Price Action: SRPT stock is trading lower by 42.0% to $21.00 during the premarket session at last check Monday.

Read Next:

• Early Wins: RegenXBio’s Gene Therapy Helps Duchenne Patients Walk Stronger, Longer

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