March 18, 2025
Dear U.S. Duchenne Community,
We are profoundly saddened to share that a young man with Duchenne muscular dystrophy has passed away following treatment with ELEVIDYS, having suffered acute liver failure. Our hearts go out to his family, friends, and everyone involved in his care.
Acute liver injury is a known possible side effect of ELEVIDYS and other AAV-mediated gene therapies and is highlighted in its prescribing information (i.e., FDA-approved label) and in the Treatment Guide for Caregivers. Although it is not a new safety signal and the risk/benefit of ELEVIDYS remains positive, acute liver failure (ALF) leading to death represents a severity of acute liver injury not previously reported for ELEVIDYS, which to date has been used to treat more than 800 clinical and prescribed patients.
In addition, testing revealed this patient had a recent cytomegalovirus (CMV) infection which was identified by the treating physician as a possible contributing factor. CMV can infect and damage the liver, a condition known as CMV hepatitis.
Patient safety and well-being are Sarepta’s top priorities. We continue to gather and analyze the information from this event. The event has been reported to the relevant health authorities and Sarepta intends to update the prescribing information to appropriately represent this event. We have also shared the event with Duchenne healthcare providers. Families should continue to consult their healthcare providers for their own clinical care decisions.
Sarepta is committed to transparency and will be sharing updated information as it becomes available with the Duchenne community.
Sincerely,
The Sarepta Patient Affairs Team
[email protected]