Douglas Ingram, president and chief executive officer of Sarepta Therapeutics Inc., during the Forbes Healthcare Summit in New York, US, on Tuesday, Dec. 5, 2023.
Michael Nagle | Bloomberg | Getty Images
Shares of Sarepta Therapeutics plunged more than 30% on Friday as the future of its approved gene therapy treatment appeared at risk.
The Food and Drug Administration will request that the company voluntarily stop all shipments of the treatment, Elevidys, a person familiar with the matter told CNBC.
Sarepta told CNBC it had not heard from the FDA.
Separately, FDA Commissioner Marty Makary told Bloomberg News the agency is considering whether the company’s gene therapy should stay on the market.
The FDA has been investigating two patient deaths tied to Elevidys. The company also reported a third death tied to a separate experimental gene therapy.
Elevidys has been mired in controversy even since before it was approved. The gene therapy has yet to clearly prove it can benefit people with Duchenne Muscular Dystrophy, a condition that erodes muscle function over time.
People with the disease eventually lose the ability to walk, and most die by their early 20s, meaning there’s a huge unmet need for treatment. The FDA in 2023 originally granted Elevidys a conditional approval for patients only between the ages of 4 and 5, the group that saw the most benefit in clinical trials.
The following year, the agency granted the treatment full approval for patients 4 and older who could still walk and accelerated approval for patients 4 and up who could no longer walk. The latter decision was especially contentious because there was less evidence that Elevidys could help people whose disease had already progressed so much.
Plus, Elevidys failed to meet its goal in a Phase 3 trial, though the company argued that the drug showed promise on other metrics in the study. Then head of the FDA’s Center for Biologics Evaluation and Research Peter Marks agreed with Sarepta’s assessment and overruled FDA staff to expand approval of Elevidys.
The company’s stock has now fallen more than 87% this year.
— CNBC’s Angelica Peebles contributed to this report
This story is developing. Please check back for updates.
