FDA Is Reportedly ‘Taking A Hard Look’ To Evaluate Whether Sarepta’s Gene Therapy Elevidys Should Remain On The Market

US Food and Drug Administration Commissioner Marty Makary said he’s “taking a hard look” at whether Sarepta Therapeutics’ gene therapy, Elevidys, should remain on the market, following recent patient deaths involving the treatment. 

Sarepta’s stock plummeted more than 26% in midday trade on Friday. However, on Stocktwits, retail sentiment around the company remained in ‘extremely bullish’ territory amid ‘extremely high’ levels of chatter.

A separate report by Reuters said that the FDA plans to request Sarepta to stop all shipments of Elevidys voluntarily.

Makary’s comments during an interview with Bloomberg come after the company on Thursday disclosed a third patient death in a clinical trial involving a different experimental gene therapy that uses a similar viral delivery system. 

A company spokesperson told Bloomberg that a 51-year-old patient died of acute liver failure last month in an early-stage trial of a gene therapy to treat limb-girdle muscular dystrophy.

The FDA had already been investigating two earlier deaths linked to Elevidys.

The growing number of fatalities is raising questions about the future of Sarepta’s therapies and the regulatory path for gene treatments that use adeno-associated viral (AAV) vectors. Elevidys, which was granted accelerated approval in 2023 for Duchenne muscular dystrophy, has remained on the market under post-approval study requirements.

Bloomberg also reported that the latest death resulted in an unusually tense exchange between analysts and Sarepta executives on a Friday call. The same group of analysts reportedly had a call with the company earlier this week about the future of Elevidys, but the firm failed to disclose the third patient death. On Friday, two of the analysts asked executives whether the company was keeping any other fatalities undisclosed.

“We are historically a very transparent organization,” said Sarepta CEO Douglas Ingram, according to the report, adding that the company didn’t disclose the third patient death earlier because “it was neither material nor central” to what was being discussed. 

Sarepta’s stock is down 88% year-to-date and nearly 90% over the past 12 months.

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