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 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,

The tragic death of the 16-year-old boy came as a result of acute liver failure, which is a known side effect of Elevidys and listed on its labelling and is thought to be linked to the adeno-associated virus (AAV) used to deliver the genetic material.