US EditionSecond patient death reported with gene therapy for muscular dystrophy
- Sarepta Therapeutics paused the use of Elevidys in non-ambulatory Duchenne muscular dystrophy patients following the fatal liver failure of a second individual receiving the therapy.
- Elevidys received accelerated FDA approval in 2023 and full expanded approval in 2024 despite limited evidence of efficacy and controversy over safety in non-ambulatory patients.
- The gene therapy, costing $3.2 million, raises dystrophin production but failed its confirmatory clinical trial and is linked to fatal liver injury in non-ambulatory patients.
- Sarepta and partner Roche paused shipments for non-ambulatory patients worldwide, plan an enhanced immunosuppressive regimen, and are working with the FDA on updated safety protocols.
- Analysts expect Elevidys will likely remain on the market with revised labeling, but the second death intensifies regulatory scrutiny and may influence physician and patient treatment decisions.
63 Articles
63 Articles
Second Patient Dies After Gene Therapy for Duchenne Muscular Dystrophy: Company
A second patient has died after receiving the gene therapy Elevidys, which was approved by the Food and Drug Administration (FDA) over concerns of staffers, according to the company that makes it. The 15-year-old boy who died, and the 16-year-old boy whose death was reported earlier in the year, had both received Elevidys, the only gene therapy approved by the FDA for the treatment of a rare form of muscular dystrophy called Duchenne muscular dy…
Sarepta reports second patient death after treatment with Duchenne gene therapy - The Boston Globe
Cambridge-based Sarepta Therapeutics said it was halting shipments of its Duchenne muscular dystrophy gene therapy for patients who can no longer walk, following the death of a second person who received the treatment.
Associated Press News
Second patient death reported with gene therapy for muscular dystrophy
Shares of Sarepta Therapeutics have plunged again after the drugmaker reported a second death in connection with its gene therapy for muscular dystrophy.
Sarepta Stops Stop Shipments of Muscular Dystrophy Gene Therapy After Second Patient Death
Like the first death reported in March, Sarepta Therapeutics attributed the fatality to a case of acute liver failure following dosing of the Duchenne muscular dystrophy gene therapy, Elevidys. The company has proposed an immunosuppressive regimen to mitigate the liver injury risk posed by the therapy. The post Sarepta Stops Stop Shipments of Muscular Dystrophy Gene Therapy After Second Patient Death appeared first on MedCity News.
Coverage Details
Bias Distribution
- 63% of the sources are Center
To view factuality data please Upgrade to Premium
