US FDA adds strongest warning to Sarepta’s Elevidys after fatal liver injuries

(Reuters) -The U.S. Food and Drug Administration said on Friday it approved new labeling for Sarepta Therapeutics’ gene therapy Elevidys that includes its most serious safety warning and restricts use of the treatment to walking patients with Duchenne muscular dystrophy.

The agency added a boxed warning to the therapy after two non-ambulatory pediatric patients died from acute liver failure following treatment. The new label removes approval for use in non-ambulatory patients entirely, limiting the drug to ambulatory patients aged four and older.

In July, Sarepta voluntarily paused distribution of the $3.2 million gene therapy for non-ambulatory patients after the FDA issued a safety communication following the deaths.

The label changes significantly narrow the patient population eligib