Angelman trial, page-93

On safety and risks in gene therapies:


Sarepta Therapeutics Shares Safety Update on ELEVIDYS
CAMBRIDGE, Mass., March 18, 2025 -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision
genetic medicine for rare diseases, shared the following safety update related to ELEVIDYS
(delandistrogene moxeparvovec-rokl), the only approved gene therapy in patients with Duchenne
muscular dystrophy.
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. Acute liver injury is a known
possible side effect of ELEVIDYS and other AAV-mediated gene therapies and is highlighted in the
prescribing information. Although it is not a new safety signal and the benefit-risk 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 patients in
clinical trials or as a prescribed therapy.
In addition, testing revealed this patient had a recent cytomegalovirus (CMV) infection which was identi-
fied 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 priority. 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
reported the event to ELEVIDYS clinical study investigators and prescribing physicians.
About ELEVIDYS (delandistrogene moxeparvovec-rokl)
ELEVIDYS (delandistrogene moxeparvovec-rokl) is a single-dose, adeno-associated virus (AAV)-based
gene transfer therapy for intravenous infusion designed to address the underlying genetic cause of
Duchenne muscular dystrophy – mutations or changes in the DMD gene that result in the lack of
dystrophin protein – through the delivery of a transgene that codes for the targeted production of
ELEVIDYS micro-dystrophin in skeletal muscle.

ELEVIDYS is indicated for the treatment of Duchenne muscular dystrophy (DMD) in individuals at least 4
years of age.
• For patients who are ambulatory and have a confirmed mutation in the DMD gene
• For patients who are non-ambulatory and have a confirmed mutation in the DMD gene.

The DMD indication in non-ambulatory patients is approved under accelerated approval based on
expression of ELEVIDYS micro-dystrophin (noted hereafter as “micro-dystrophin” in skeletal muscle.
Continued approval for this indication may be contingent upon verification and description of clinical
benefit in a confirmatory trial(s).