The third death linked to Sarepta’s gene therapy programs—following two fatalities in Elevidys-treated DMD patients and one in an investigational LGMD trial—has triggered heightened regulatory action and intensified industry scrutiny. The FDA has halted related trials, revoked platform designation, and is pressing Sarepta to pause Elevidys distribution. Acute liver failure remains the central safety concern, underscoring urgent questions about the use of AAVrh74-based therapies.
Elevidys’ future remains uncertain, as regulators contemplate a market withdrawal or tighter restrictions, while Sarepta braces for fallout in investor trust and examines broader strategic shifts.
SHARE
