FDA Revises Elevidys Label With Boxed Warning, Limits Use to Ambulatory Duchenne Patients
The U.S. Food and Drug Administration (FDA) announced on November 14 that it has approved revised labeling for the gene therapy Elevidys, adding a boxed warning and limiting the indication to ambulatory patients with Duchenne muscular dystrophy (DMD). The revision follows reports of fatal acute liver failure in two non-ambulatory pediatric male patients.
The FDA said the updated label carries the agency's most serious boxed warning, alerting that the therapy can cause serious liver injury and acute liver failure, including fatal outcomes. The revised indication covers patients aged 4 years and older with a confirmed mutation in the DMD gene who are able to walk; the indication for non-ambulatory patients has been removed. The agency had reported the two fatal cases in a June 2025 safety communication.
Under the new label, liver function should be tested weekly for at least three months after dosing, patients should remain close to a medical facility for at least two months, and troponin-I should be monitored weekly for one month. The therapy is contraindicated in patients with deletions of exon 8 and/or exon 9 in the DMD gene. The FDA is also requiring a postmarketing observational study of about 200 patients followed for at least 12 months to assess liver-related safety.
Source: U.S. Food and Drug Administration
Published by GSM · USA (original Chinese edition)
Photo: 图中是美国食品药品管理局（FDA）总部入口。Wikimedia Commons / U.S. Food and Drug Administration, Public domain
