In November 2025 FDA added a Boxed Warning for serious liver injury and acute liver failure to Elevidys and restricted the indication to ambulatory patients aged four years and older after fatal acute liver-failure reports in non-ambulatory patients.

What this does not establish

Prior approval was not permanent proof of safety; post-market restriction does not quantify risk for every subgroup or prove a class-wide rate.

Counterevidence & uncertainty

Exposure denominators and causal mechanisms remain important for absolute-risk estimation.

What would change the reading

Update with final investigations, denominator-based rates, mechanistic evidence and further label action.

Primary routes

1. BL-S017https://www.fda.gov/news-events/press-announcements/fda-approves-new-safety-warning-and-revised-indication-limits-use-elevidys-following-reports-fatalOpen source record →