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FDA Adds Boxed Liver Warning and Limits Elevidys to Ambulatory Boys 4 and Up

The decision also requires intensive monitoring with a 200‑patient study to clarify liver risk after postmarket deaths.

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Elevidys has been approved for use in ambulatory patients aged four years and older who have confirmed mutations in the DMD gene. Credit: MY STOCKERS/Shutterstock.com.
US FDA Restricts Duchenne Gene Therapy Following Fatal Liver Failure Cases in Teens

Overview

  • Regulators removed non‑ambulatory patients from the indication, restricting use to ambulatory patients aged four years and older with confirmed DMD mutations.
  • The updated label carries a boxed warning for serious liver injury, acute liver failure, and death.
  • The FDA linked two teen fatalities to Elevidys following sharply elevated liver enzymes and hospitalization within two months of infusion, with three post‑treatment deaths reported overall and one nonfatal acute liver injury case.
  • Prescriber guidance now recommends a modified corticosteroid regimen, weekly liver tests for at least three months, weekly cardiac testing for one month, and warns of higher infection risk due to immunosuppression.
  • Sarepta previously paused shipments and plans to study an enhanced sirolimus immunosuppression regimen to address liver risk and seek restored access for non‑ambulatory patients, noting more than 1,100 patients have received the therapy worldwide.