Drugmaker refuses FDA request to pull gene therapy
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An unusual public feud between the Food and Drug Administration and a maker of gene therapies for rare diseases could test the Trump administration's willingness to pull certain approved drugs from the market over safety concerns.
The big picture: The standoff with Cambridge, Massachusetts-based Sarepta Therapeutics burst into public view late Friday after the company refused an FDA request to withdraw its treatment for Duchenne muscular dystrophy following patient deaths from liver failure.
- Duchenne is a rare genetic condition often detected in young boys that leads to wasting away of the body's muscles and often kills patients in their 20s or 30s.
Driving the news: The FDA said agency leadership met with Sarepta on Friday and requested it voluntarily stop all shipments of the IV therapy, called Elevidys, that day. The company refused.
- Sarepta said it would pause shipments for patients confined to wheelchairs but would continue shipments to ambulatory patients, saying there were no new or changed safety signals in that group.
- The company's stock plummeted on the news, while FDA commissioner Marty Makary told Bloomberg his agency was considering whether the therapy should continue to be sold.
Between the lines: The FDA gave Elevidys an "accelerated approval" in 2023 using a fast-track process that lets drugmakers sell their products based on preliminary evidence.
- It later expanded the approval to more patients, despite the failure of a clinical trial to meet its primary goal of improving patients' motor abilities, per the New York Times.
- The agency last month began investigating cases of liver failure connected with Elevidys after receiving reports of two wheelchair-bound pediatric patient deaths.
- Last week, Sarepta reported a third death of an older patient in a clinical trial for a different gene therapy. That news came a day after the company announced a restructuring and laid off more than a third of its workforce.
- The gene therapies are different but are delivered in the same way. That's increased safety concerns, given the uncertain benefit of the Duchenne drug, BMO analyst Kostas Biliouris told CNBC.
What they're saying: "We are committed to working closely with the FDA to ensure that all decisions are grounded in science and the best interests of patients, considering the compelling need of these families to access disease-modifying therapy," Sarepta said in a statement.
- Makary said Friday's request for the company to stop all shipments of Elevidys showed that "We believe in access to drugs for unmet medical needs but are not afraid to take immediate action when a serious safety signal emerges."
What's next: The FDA could seek to pull the approval if it concludes safety risks outweigh the treatment's benefits.
- The agency made such a move in 2023, when it withdrew a controversial premature birth drug that had been on the market for more than a decade.
