Back to today’s briefing

Story perspectives

FDA Investigates Fatalities Linked to Sarepta's Gene Therapy

6/26/2025

25 4

Summary

Story summary

The FDA is probing the tragic deaths of two boys from liver failure linked to Sarepta's gene therapy, Elevidys, used for Duchenne muscular dystrophy. In response to the second death, Sarepta has paused Elevidys treatments for non-ambulatory patients. Stricter safety warnings or a potential withdrawal of its accelerated approval loom on the horizon.

1 of 1

Story summary

The FDA is probing the tragic deaths of two boys from liver failure linked to Sarepta's gene therapy, Elevidys, used for Duchenne muscular dystrophy. In response to the second death, Sarepta has paused Elevidys treatments for non-ambulatory patients. Stricter safety warnings or a potential withdrawal of its accelerated approval loom on the horizon.