Safety Communication on 2 Elevidys-related Deaths

FDA has issued a safety communication about two reports of fatal acute liver failure following treatment of non-ambulatory pediatric male Duchenne Muscular Dystrophy patients with Sarepta Therapeutics’ Elevidys (delandistrogene moxeparvovec-rokl), an adeno-associated virus vector-based gene therapy. The safety alert comes after the company said last week it had stopped shipping the gene therapy and paused dosing in a key confirmatory trial after the second death was reported (see earlier story).

The agency says that in both cases, the patients presented with elevated transaminases and were hospitalized less than two months after treatment. “FDA is investigating the risk of acute liver failure with serious outcomes, including those such as hospitalization and death, following Elevidys, and is evaluating the need for further regulatory action,” it says.