Patient Passes Away After Gene Therapy for Duchenne Muscular Dystrophy
A patient experienced acute liver failure after receiving Elevidys treatment, with Sarepta Therapeutics attributing a preexisting infection as a potential contributing factor.
“We are deeply saddened to report that a young man with Duchenne muscular dystrophy has lost his life following treatment with ELEVIDYS, after suffering from acute liver failure,” stated the company in its report.
“The safety and well-being of our patients is Sarepta’s primary concern. We are actively collecting and analyzing data regarding this incident,” the company added.
Sarepta Therapeutics, a leading entity in genetic medicine, acknowledged that while acute liver injury is a recognized risk associated with Elevidys and other “AAV-mediated gene therapies,” the occurrence of acute liver failure leading to fatality had not been previously documented among the over 800 patients who have received the therapy.
In its statement, Sarepta mentioned that the patient had also recently been diagnosed with cytomegalovirus (CMV), which the treating physician suggested might be a contributing factor to his health condition. CMV can impact the liver and result in CMV hepatitis, potentially increasing the risks linked to gene therapy, according to the company.
Sarepta has reported this incident to the relevant health authorities and plans to update Elevidys’ prescribing information to reflect the seriousness of this adverse event. The company has also informed clinical study investigators and prescribing healthcare professionals about the situation.
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Elevidys, also referred to as delandistrogene moxeparvovec-rokl, is the sole approved gene therapy for Duchenne muscular dystrophy (DMD), a genetic neuromuscular disorder that primarily affects young boys and leads to significant muscle degeneration and weakness.
The treatment utilizes a modified virus to deliver a replacement gene to enable the production of dystrophin within patient cells. It is priced at approximately $3.2 million for a single treatment, making it one of the most costly pharmaceuticals globally.
Sarepta has encouraged healthcare providers to closely monitor liver function in patients treated with Elevidys for a minimum of three months post-infusion. The company also reiterated its advice for the use of systemic corticosteroids both before and after the therapy to minimize liver-related risks.
The FDA has not taken any new regulatory actions regarding Elevidys following this report.
The Associated Press contributed to this report.
