• Posted March 19, 2025

Sarepta’s Gene Therapy Under Scrutiny After Patient Death

A patient has died while receiving Elevidys, a gene therapy for Duchenne muscular dystrophy, marking the first known death linked to the treatment.

The company behind the drug, Sarepta Therapeutics, said that the patient suffered acute liver injury, a known side effect, The Associated Press reported. However, the "severity" of this case had not been seen before, the company noted. Elevidys has been used in more than 800 patients, the company stated.

Sarepta did not release details about the patient but said a recent infection may have played a role in the liver injury. The therapy has been used in more than 800 patients, the company said.

The company plans to update the prescribing information for Elevidys to reflect this case.

Shares of Sarepta, based in Cambridge, Massachusetts, dropped more than 27% on Tuesday, closing at about $73 per share.

Elevidys, which costs $3.2 million for a one-time infusion, was the first gene therapy approved in the U.S. for Duchenne muscular dystrophy, a rare condition that causes muscle weakness, mobility loss and early death in males.

The FDA first granted the drug expedited approval in 2023, despite some scientists questioning its effectiveness.

In 2024, the agency expanded full approval to include patients 4 and older, regardless of mobility status.

The therapy works by using a disabled virus to insert a gene that helps the body produce dystrophin, a protein crucial for muscle function. Sarepta said more than 800 patients have received Elevidys so far, The Associated Press reported.

Sarepta has secured FDA accelerated approval for three other Duchenne drugs since 2016. But none have yet been confirmed to be effective and further studies are still ongoing.

More information

Learn more about Elevidys.

SOURCE: The Associated Press, March 18, 2025