Sarepta , Roche halt DMD gene therapy Elevidys for non-ambulatory patients

USA—Sarepta Therapeutics and its global partner Roche have announced an immediate halt to the commercial and clinical use of their Duchenne muscular dystrophy (DMD) gene therapy, Elevidys (delandistrogene moxeparvovec), in non-ambulatory patients.

This decision follows a safety re-assessment after two non-ambulatory patients treated with Elevidys died from acute liver failure (ALF), a serious side effect linked to the therapy.

Roche has stopped commercial dosing of Elevidys in non-ambulatory patients worldwide and paused enrollment and dosing in clinical trials for this group until new risk mitigation strategies, such as enhanced immune suppression, are implemented.

Meanwhile, Sarepta is suspending shipments of Elevidys for non-ambulatory patients in the U.S. while it convenes an independent panel of Duchenne and liver health experts to evaluate an improved immunosuppressive regimen aimed at reducing liver toxicity.

Elevidys is currently the only gene therapy approved in the U.S. for treating DMD patients aged four and older, including both ambulatory and non-ambulatory individuals.

It works by delivering a functional copy of the DMD gene to produce dystrophin, a protein essential for muscle strength and protection.

Duchenne muscular dystrophy is a rare, progressive genetic disease primarily affecting boys, leading to muscle degeneration, loss of mobility, and severe complications such as respiratory and heart failure. The average life expectancy for those affected is about 28 years.

The first fatal ALF case linked to Elevidys was reported in March 2025, prompting the suspension of multiple clinical trials.

The second death, announced in June 2025, involved another non-ambulatory patient and has led to further restrictions and pauses in treatment.

Both companies have reported these events to the U.S. Food and Drug Administration (FDA) and other global health authorities but have not released detailed information about the second death.

Financially, the news impacted the companies’ stocks, with Roche shares dropping 1% at market open and Sarepta’s shares plunging over 31% in pre-market trading on the Nasdaq.

Despite these setbacks, analysts remain cautiously optimistic. William Blair analyst Sami Corwin noted that while another patient death raises concerns about Elevidys’ future, the likelihood of the therapy being fully withdrawn from the market remains low.

However, the drug’s label is expected to be updated to include warnings about the risk of acute liver failure.

Sarepta’s CEO and chief scientific officer emphasized that patient safety is the highest priority and that the company is taking urgent steps to better understand and mitigate liver-related risks, including the potential addition of the immunosuppressant sirolimus to the treatment regimen.

This drug is intended to manage liver toxicity but carries its own risks, such as increased infection susceptibility.

Sarepta is actively engaging with the FDA to update Elevidys’ clinical protocols accordingly.

Elevidys has been approved by multiple regulatory authorities worldwide, including in the U.S., Japan, and several Middle Eastern countries.

To date, over 900 patients have received the therapy through clinical trials and real-world use.

Roche and Sarepta jointly manage Elevidys’ development and clinical studies, with Roche holding rights outside the United States and Sarepta holding U.S. rights.

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