FDA Suspends Muscular Dystrophy Drug After 3 Patient Deaths

The U.S. Food and Drug Administration (FDA) has placed an immediate clinical hold on Sarepta Therapeutics’ investigational gene therapy trials for limb girdle muscular dystrophy following three patient deaths potentially linked to the company’s treatments.

The agency announced the action on Friday, citing serious safety concerns and “unreasonable and significant risk” to study participants. The FDA also revoked the company’s platform technology designation and requested Sarepta voluntarily halt shipments of its approved drug Elevidys, which the company refused to do.

Newsweek reached out to Sarepta Therapeutics via email on Sunday after normal business hours for comment.

Why It Matters

This regulatory intervention represents a significant safety measure in the gene therapy field, affecting both ongoing clinical trials and an already approved treatment for Duchenne muscular dystrophy.

The decision could impact treatment options for families dealing with these devastating muscle-wasting diseases. Duchenne muscular dystrophy is a rare and serious genetic condition that worsens over time, leading to weakness and wasting away of the body’s muscles.

What To Know

The three deaths resulted from acute liver failure in patients treated with either Elevidys or investigational therapies using the same AAVrh74 viral vector technology. One fatality occurred during a clinical trial for limb girdle muscular dystrophy treatment.

Elevidys is an adeno-associated virus vector-based gene therapy designed to deliver a gene that leads to production of a shortened protein that contains selected domains of the dystrophin protein present in normal muscle cells. The disease occurs due to a defective gene that results in abnormalities in, or absence of, dystrophin, a protein that helps keep the body’s muscle cells intact.

The drug received traditional approval for ambulatory DMD patients aged 4 and older on June 20, 2024, while maintaining accelerated approval for non-ambulatory patients since June 22, 2023. Under accelerated approval, companies must conduct confirmatory studies to verify clinical benefit. Given the new safety concerns, the FDA has notified Sarepta that the drug’s indication should be restricted to ambulatory patients only.

The FDA revoked the platform technology designation for Sarepta’s AAVrh74 Platform Technology because the preliminary evidence is insufficient to demonstrate that the technology has the potential to be incorporated in multiple drugs without adverse safety effects.

What People Are Saying

FDA Commissioner Marty Makary, M.D., M.P.H.: “Today, we’ve shown that this FDA takes swift action when patient safety is at risk. We believe in access to drugs for unmet medical needs but are not afraid to take immediate action when a serious safety signal emerges.”

Dr. Vinay Prasad, Director of FDA’s Center for Biologics Evaluation and Research: “Protecting patient safety is our highest priority, and the FDA will not allow products whose harms are greater than benefits. The FDA will halt any clinical trial of an investigational product if clinical trial participants would be exposed to an unreasonable and significant risk of illness or injury.”

What Happens Next

The FDA will continue investigating the risk of acute liver failure and other serious outcomes following gene therapies using Sarepta’s AAVrh74 platform technology. The agency has committed to taking additional regulatory actions as needed to protect patients.

For current Elevidys patients, the FDA is focusing its safety investigation on ambulatory patients while restricting the drug’s use in non-ambulatory patients.