Shares young man with Duchenne Muscular Dystrophy 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
Tuesday, March 18, 2025 8:01:43 AMEST
- 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 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 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.
**Note: The median age of survival in males with DMD was 23.7 years. Duchenne muscular dystrophy (DMD) patients are at risk of acute liver failure. Children with neuromuscular disorders, including DMD, are also at risk of liver enlargement, steatosis, and fibrosis. Despite medical advances, some DMD patients die prematurely, and prognostic factors for worse outcomes have been identified.
*Reminder: 03/17 CAPR Ongoing HOPE-2 open label extension clinical trial demonstrates potential of deramiocel to slow disease progression and preserve upper limb function in patients with Duchenne muscular dystrophy (“DMD”)
*Reminder: 12/18/24 SRPT Completes enrollment in EMERGENE, a phase 3 clinical study of SRP-9003 for treatment of limb-girdle muscular dystrophy type 2E/R4; Data from EMERGENE expected in 1H25 with BLA submission to US FDA, seeking accelerated approval, for SRP-9003 in 2025
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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 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 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.
**Note: The median age of survival in males with DMD was 23.7 years. Duchenne muscular dystrophy (DMD) patients are at risk of acute liver failure. Children with neuromuscular disorders, including DMD, are also at risk of liver enlargement, steatosis, and fibrosis. Despite medical advances, some DMD patients die prematurely, and prognostic factors for worse outcomes have been identified.
*Reminder: 03/17 CAPR Ongoing HOPE-2 open label extension clinical trial demonstrates potential of deramiocel to slow disease progression and preserve upper limb function in patients with Duchenne muscular dystrophy (“DMD”)
*Reminder: 12/18/24 SRPT Completes enrollment in EMERGENE, a phase 3 clinical study of SRP-9003 for treatment of limb-girdle muscular dystrophy type 2E/R4; Data from EMERGENE expected in 1H25 with BLA submission to US FDA, seeking accelerated approval, for SRP-9003 in 2025