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2mo ago

Regulators Halt Elevidys Gene Therapy Worldwide After Safety Failures

Authorities around the world have suspended the treatment after patients suffered fatal liver failure.

Cayse Blair, who has Duchenne muscular dystrophy, was supposed to be dosed with a gene therapy this summer before the FDA asked it be pulled from the market.
MIAMI, FL - MARCH 23: Bottles of antidepressant pills Wellbutrin (L-R) , Paxil, Lexapro, Effexor, Zoloft and Fluoxetine are shown March 23, 2004 photographed in Miami, Florida. The Food and Drug Administration asked makers of popular antidepressants to add or strengthen suicide-related warnings on their labels as well as the possibility of worsening depression especially at the beginning of treatment or when the doses are increased or decreased. (Photo Illustration by Joe Raedle/Getty Images)
A sign marks the offices of Sarepta Therapeutics in Cambridge, Massachusetts, U.S., July 22, 2025. REUTERS/Brian Snyder/File Photo
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Overview

  • Regulators in the United States, Europe, Japan and Brazil have halted all use of Elevidys, leaving the gene therapy off-market globally.
  • The EMBARK phase 3 trial failed to meet its primary endpoint, showing no significant improvement in ambulation scores after 12 months.
  • Two teenage patients treated with Elevidys died from acute liver failure, triggering the FDA’s shipment halt request and safety reviews.
  • Sarepta reversed its initial refusal and voluntarily paused U.S. shipments on July 21, while Roche plans to engage the EMA on possible reauthorization.
  • Duchenne families nationwide report confusion and heartbreak as scheduled infusions are postponed indefinitely.