Sarepta Pauses European DMD Gene Therapy Trials After Patient Death
April 08, 2025
Sarepta Therapeutics has
temporarily paused recruitment and dosing in select clinical trials of delandistrogene moxeparvovec-rokl (Elevidys), the company’s
FDA-approved gene therapy for Duchenne muscular dystrophy, following a request from European Union reference member state authorities. The pause follows Sarepta’s March 18 safety update regarding a
case of acute liver failure (ALF) in a patient who received the treatment.
On April 3, an independent data monitoring committee (DMC) met to evaluate the event and determined that the overall benefit-risk profile remains favorable. The DMC also recommended that dosing continue without changes to study protocols. Sarepta and its partner Roche will submit these findings to EU regulators within a week.
In its
safety update, Sarepta noted that while ALF is a known potential side effect of adeno-associated virus (AAV)-mediated gene therapies, but ALF leading to death represents a severity of acute liver injury not previously reported. The company also plans to update the prescribing information to reflect this event.
