The commercial and clinical use of Elevidys was discontinued after two fatal cases of acute liver failure.

Children’s Hospital Los Angeles paused the use of Sarepta Therapeutics Inc.’s gene therapy for Duchenne muscular dystrophy in the wake of rising safety concerns about liver toxicity, adding to mounting challenges for the drugmaker.

The regulator had asked Sarepta Therapeutics to halt all shipments of its therapy, Elevidys, after three patients died from liver failure after taking it or a similar treatment.

Sarepta Therapeutics faces an "arduous path” to try to get its gene therapy for Duchenne muscular dystrophy back onto the market, a senior FDA official told STAT.

Sarepta Therapeutics Inc. said another patient has died from acute liver failure after receiving one of its gene therapies, putting additional pressure on the biotech company after the recent deaths of two teenage boys.

Sarepta Therapeutics will cut 500 jobs and add a serious warning on the label of its muscle-disorder gene therapy Elevidys, the drugmaker said on Wednesday, following the recent deaths of two patients who were on the medicine.