Patient Dies Following Muscular Dystrophy Gene Therapy

Introduction to the Incident

By MATTHEW PERRONE, AP Health Writer

WASHINGTON (AP) — Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, sending company shares plummeting in morning trading.

Details of the Incident

The young man died of acute liver injury, a known side effect, Sarepta said in a statement. But the company said the “severity” of the patient’s case had not previously been seen with the therapy, called Elevidys. It’s the first known patient death with the therapy, which has been used in more than 800 patients, the company said.

Background on Elevidys

In 2023, Elevidys received expedited U.S. approval despite concerns from some Food and Drug Administration scientists about its effectiveness in treating Duchenne muscular dystrophy. It’s the first gene therapy approved in the U.S. for the rare muscle-wasting condition, which causes weakness, loss of mobility and early death in males.

The FDA granted full approval last year for Duchenne’s patients with a particular genetic mutation, expanding its use to patients 4 and older, regardless of whether they are still able to walk. Previously it was only available for younger patients who were still mobile.

Reaction from Sarepta

Sarepta said in a statement the patient who died had a recent infection that could have contributed to the liver injury. The company said it plans to update the prescribing information for Elevidys to reflect the case.

Impact on Sarepta

Shares of the Cambridge, Massachusetts-based company fell more than 23% to about $78 per share in morning trading.

How Elevidys Works

Elevidys uses a disabled virus to insert a replacement gene for producing dystrophin into patient cells. It costs $3.2 million for a one-time treatment.

Sarepta’s History with Duchenne’s Drugs

Sarepta has received FDA accelerated approval for three other Duchenne’s drugs since 2016. None has yet been confirmed to work; studies designed to secure full FDA approval are ongoing.

Conclusion

The incident highlights the risks associated with gene therapy, even as it offers hope for treating debilitating conditions like muscular dystrophy. The death of a patient due to a known side effect underscores the need for continued vigilance and research into the safety and efficacy of such treatments.

FAQs

• Q: What is Elevidys used for?
  A: Elevidys is used for the treatment of Duchenne muscular dystrophy, a rare muscle-wasting condition.
• Q: How much does Elevidys cost?
  A: Elevidys costs $3.2 million for a one-time treatment.
• Q: What happened to the patient who was taking Elevidys?
  A: The patient died due to acute liver injury, a known side effect of the therapy.
• Q: How has the FDA been involved with Elevidys?
  A: The FDA granted expedited approval for Elevidys in 2023 and full approval last year for patients with a particular genetic mutation.
• Q: What is Sarepta’s history with Duchenne’s drugs?
  A: Sarepta has received FDA accelerated approval for four Duchenne’s drugs since 2016, including Elevidys.

  Originally Published: March 18, 2025 at 11:36 AM PDT

  The Associated Press Health and Science Department receives support from the Howard Hughes Medical Institute’s Science and Educational Media Group and the Robert Wood Johnson Foundation. The AP is solely responsible for all content.