FDA adds boxed warning to Duchenne gene therapy
The FDA on Nov. 14 added a boxed warning to Sarepta Therapeutics' Elevidys gene therapy for Duchenne muscular dystrophy after two patient deaths and limited its approved use to ambulatory patients age 4 and older. New labeling also recommends weekly liver-function monitoring for the first three months post-infusion and other precautions, affecting how Twin Cities providers prescribe and monitor the one-time treatment.
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📌 Key Facts
- FDA issued a boxed warning for potentially fatal liver failure with Elevidys
- Use narrowed to ambulatory patients aged 4+ (no longer for non‑ambulatory patients)
- Label advises weekly liver tests for three months after infusion
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November 14, 2025