We are deeply saddened to learn of the passing of a young man who received ELEVIDYS gene therapy for Duchenne muscular dystrophy. Our hearts go out to his family during this incredibly difficult time.
Tragically, the young man (aged 16) passed away after suffering acute liver failure. While acute liver injury is a recognised potential side effect of ELEVIDYS and similar gene therapies using AAV vectors, it is not considered a new safety signal and the risk/benefit of delandistrogene moxeparvovec remains unchanged. Acute liver failure (ALF) leading to death represents a severity of acute liver injury not previously reported for delandistrogene moxeparvovec.
Subsequent tests showed the patient had a recent cytomegalovirus (CMV) infection. The treating physician identified this infection as a possible contributing factor, as CMV can cause liver damage (CMV hepatitis).
Sarepta/ Roche have reiterated that patient safety and well-being remains their highest priority, with the companies continuing to gather and analyse information from this event.
We understand this news will be deeply upsetting for many within the Duchenne community. Action Duchenne is here to provide support and answer any questions you may have. Please do not hesitate to reach out to us at info@actionduchenne.org.
We are closely monitoring the situation and will share any relevant updates as they become available.
For more information, please see the statement released by Sarepta following this sad news.
What is ELEVIDYS?
ELEVIDYS, also known as delandistrogene moxeparvovec (SRP-9001), is a gene therapy designed to address the root cause of Duchenne muscular dystrophy (DMD). It’s a single-dose treatment that utilises an adeno-associated virus (AAV) vector to introduce a functional version of the dystrophin gene into the cells of patients who have a confirmed genetic mutation causing DMD.
EMBARK
A Phase 3 Multinational, Randomised, Double-Blind, Placebo-Controlled Systemic Gene Delivery Study to Evaluate the Safety and Efficacy of SRP-9001 in Patients With Duchenne Muscular Dystrophy. The study will evaluate the safety and efficacy of gene transfer therapy in 120 ambulatory boys aged between 4 and 7 with DMD.
