Elevidys was approved after intense debate at the FDA, as officials and reviewers disagreed over how strong the evidence was and whether it warranted making the drug available for patients facing a fatal disease with few other options.

A young man treated with Elevidys died of acute liver failure. The case may give doctors pause before prescribing the treatment in the future, some analysts wrote.

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.