FDA to review expanded use of Sarepta Duchenne gene therapy

Dive Brief:

• The Food and Drug Administration will decide by June 21 whether to loosen the restrictions surrounding use of Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy, the company said Friday
• Sarepta’s gene therapy, Elevidys, was granted a conditional OK last June for children between 4 and 5 years of age who have Duchenne and can still walk. Sarepta aims to convert that nod to a full approval for all people with Duchenne and a confirmed mutation to a specific gene, even though Elevidys failed to hit its main goal in a study meant to confirm its benefits. 
• Sarepta, for its part, has argued that the collective evidence it’s accrued proves Elevidys is impacting the disease. Analysts appear optimistic of its chances, given the agency won’t convene a group of outside experts to review its request.  

Dive Insight:

The announcement is a boost for Sarepta, which has been able to bring multiple Duchenne drugs to market without definitive evidence they can change the course of the disease. Elevidys was among them, and the FDA’s willingness to consider expanding use without first asking outside experts to weigh in is a “positive sign for expansion and conversion to full approval,” wrote William Blair analyst Tim Lugo, in a note to investors. 

Sarepta’s shares rose by 10% in morning trading Friday, reaching levels it hasn’t traded at since last June. 

In climbing back that far, the company has fully recovered the value lost when it announced that Elevidys had failed its confirmatory trial. Sarepta argued at the time that, though Elevidys failed to show a statistically significant benefit over a placebo in a measure of motor function, a variety of other data points proved it helped study participants. The “totality of the evidence” persuaded FDA officials to consider expanding the drug’s use, CEO Doug Ingram said at the time.

The agency’s decision to, at this point, forego an advisory committee could be taken as a strong endorsement. But the FDA did reverse course with Sarepta’s initial Elevidys application, holding a meeting after initially communicating to the company that it wouldn’t. 

“We acknowledge that anything is possible,” Leerink Partners analyst Joseph Schwartz wrote in a research note.

Notably, Sarepta aims to broaden use of Elevidys to include patients who can no longer walk, a group whose disease is more advanced. That request “is more of a stretch,” Lugo wrote. “We would not be surprised if these patients are excluded from the expanded label.”

Ingram told attendees at the J.P. Morgan Healthcare Conference in January that Elevidys recorded sales of $200 million in 2023. The company has not formally announced fourth-quarter and full-year results.

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