FDA修订Elevidys说明书：新增黑框警告，用药限于可行走的杜氏肌营养不良患者
美国食品药品管理局（FDA）于2025年11月14日宣布，批准修订基因疗法Elevidys的说明书，新增黑框警告，并将适应证限于可行走的杜氏肌营养不良症（DMD）患者。此次修订源于两例无法行走的男性患儿发生致命性急性肝衰竭的报告。
FDA表示，新版说明书加入该机构最高级别的黑框警告，提示该药存在严重肝损伤和急性肝衰竭风险，包括致命结局。修订后，该药适用于4岁及以上、经确诊带有DMD基因突变且能够行走的患者；用于无法行走患者的适应证已被移除。FDA曾在2025年6月的安全通报中报告上述两例死亡病例。
根据新版说明书，患者在给药后至少三个月内应每周接受肝功能检测，给药后至少两个月内应留在医疗机构附近，给药后一个月内每周监测肌钙蛋白I。带有DMD基因第8和/或第9外显子缺失的患者禁用该药。FDA同时要求开展一项上市后观察性研究，纳入约200名患者，随访至少12个月，以评估肝脏相关安全性。
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.
来源：美国食品药品管理局
发布：GSM·美国（发布链接）
图片来源：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
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