Sarepta, Reports 16 Years Old Boy Death After Elevidys Gene Therapy Treatment

Sarepta Therapeutics said on Tuesday a young man has died due to acute liver failure after treatment with its gene therapy (Elevidys) for a rare muscular dystrophy.

On Tuesday, Sarepta Therapeutics said that the first recorded fatality associated with their gene therapy for Duchenne muscular dystrophy, Elevidys, had occurred in a 16-year-old boy.

Teen Who Received Elevidys Infusion Dies

Over the past 20 years, several gene therapy procedures have been known to cause liver failure, which is what happened to the youngster. [Read More: Frequently Asked Questions About Elevidys Used for Duchenne Muscular Dystrophy]

The death “represents a severity of acute liver injury not previously reported for Elevidys,” Sarepta noted. The patient had also tested positive for cytomegalovirus (CMV) infection, which can damage the liver and is “a possible contributing factor” to the death, according to the announcement. Sarepta expressed “profound” sadness over the death. [What is Cytomegalovirus Infection (CMV)?]

Sarepta Shares Drop 28%

Sarepta has reported the death to the relevant health authorities and will update Elevidys’ label to “appropriately represent this event,” the biotech noted in its Tuesday release. The company’s stock plunged around 28% as the markets opened on Tuesday to $73.01, down from $101.35 at the previous market close.

Learn More: Clinical Trials Review of Elevidys Gene Therapy: Is Elevidys Cost Worthy?

In Which Country and In Which Hospital Did This Sad Incident Occur?

It was reported by Sarepta that the young DMD patient who died was a US citizen and the incident occurred in the US. [Read more: Community Letter]

If sufficient and satisfactory information comes from Sarepta, we will notify all DMD communities that we will make a special news on this subject.

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