The news: Rare disease drugmaker Sarepta Therapeutics is pausing sales of a muscular dystrophy drug, Elevidys, after initially refusing an FDA request to halt sales due to safety concerns.

A Duchenne muscular dystrophy (DMD) patient died in June from acute liver failure while taking Elevidys. This followed another similar death in March. Just last week, Sarepta said a third patient in a clinical trial also died of liver failure in June.

How we got here: The gene therapy drug was initially approved by the FDA in 2023 for ambulatory DMD patients ages 4-5 with a specific gene expression. An accelerated approval for non-ambulatory patients ages 4 and older was added in June 2024.

Both FDA approvals were controversial, as they were based on early clinical trial results and greenlit by then-CBER regulatory agency chief Peter Marks, who overruled FDA expert staff recommendations to reject the drug. Parent and patient advocates desperate for treatments played a key role in advocating for Elevidys’ approval. DMD is a genetic disease that causes progressive muscle loss in boys beginning in childhood. The prognosis for individuals with DMD is an average life expectancy of about 30 years. The only previous treatments were steroids to delay muscle weakness.

Zooming out: It’s unclear what happens next. Although the three deaths occurred in non-ambulatory and older patients, the 4-to-5-year-olds who could get the drug under the initial approval are also locked out for now. A group of mothers of sons with DMD wrote an emotional op-ed in STAT last week detailing their heartbreak, but also willingness to accept the risks of the drug versus the risk of no treatment.

The takeaway: Elevidys’ path to FDA approval was unconventional, but deemed appropriate at the time for a devastating disease. The new deaths and now paused sales serve as caution for both the FDA and drugmakers to balance the emotional sway of unmet medical needs with complete and continued clinical research.

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