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2h ago

FDA Adds Boxed Liver Warning to Elevidys, Restricts Use to Ambulatory Duchenne Patients

The decision follows FDA findings linking the gene therapy to fatal cases of acute liver injury.

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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.

Overview

  • Updated U.S. prescribing information now limits Elevidys to boys aged 4 and older who can walk and have confirmed DMD mutations.
  • The label carries a boxed warning for acute liver failure and injury, with new guidance for weekly liver tests for three months and weekly cardiac checks for one month after infusion.
  • Regulators attributed two of three reported post-treatment deaths to Elevidys following severe liver injury, and a nonfatal acute liver injury case has also been reported.
  • The FDA is requiring a 200-person, 12-month study to better define serious hepatic risk, and the label adds a warning about susceptibility to serious infections from immunosuppression.
  • Prescriber instructions include a modified corticosteroid regimen around infusion, and Sarepta plans a study of enhanced sirolimus immunosuppression as it seeks a path to resume dosing in non-ambulatory patients pending FDA review.