Nov. 14 at 8:54 PM
Sarepta Therapeutics shares rose after the FDA approved new labeling for its gene therapy Elevidys, adding a prominent safety warning and restricting use to ambulatory Duchenne muscular dystrophy patients aged four and older. The move follows reports of fatal acute liver failure in non-ambulatory children treated with the therapy. Investors welcomed the decision, viewing it as a sign the FDA would limit—rather than withdraw—Elevidys’ authorization.
The revised label removes the indication for non-ambulatory DMD patients and adds the agency’s strongest warning. It also requires weekly liver-function tests for at least three months post-treatment and recommends patients stay near appropriate medical facilities for two months after infusion. The FDA is mandating a post-marketing observational study of about 200 DMD patients to further assess the risk of severe liver injury.
$SRPT