Sarepta Therapeutics has refused a request from the U.S. Food and Drug Administration (FDA) to pause shipments of its gene therapy Elevidys following the deaths of three patients, including two teenage boys with Duchenne muscular dystrophy (DMD) and one adult in a related trial.
The FDA revealed that the two teenage boys, both unable to walk, died from acute liver failure after receiving Elevidys. Another patient, a 51-year-old man enrolled in a Sarepta trial for a different gene therapy targeting limb-girdle muscular dystrophy (LGMD), also died last month from acute liver failure.
After these incidents, FDA officials met with Sarepta and asked the company to voluntarily stop shipments of Elevidys, which makes up more than half of Sarepta’s product revenue. Sarepta declined the request, citing its own scientific review indicating no new safety concerns among patients who are still ambulatory.
Earlier in June, Sarepta had already suspended Elevidys shipments for non-ambulatory patients, who are at higher risk. The company said that approximately 85% of treated boys could still walk.
FDA Commissioner Marty Makary said the agency is actively reviewing whether Elevidys should remain available on the market.
The recent death involving the LGMD gene therapy, which uses the same viral delivery system as Elevidys, has raised wider safety concerns. Analyst Brian Skorney noted that the risk of the FDA fully removing Elevidys from the market has increased.
News of the FDA’s request to halt shipments was first reported by Reuters and Bloomberg on Friday. Sarepta’s shares fell sharply, closing down over 30% after the announcement.
The gene therapies use an adeno-associated virus (AAV) vector, which Sarepta has previously linked to fatal liver complications. Critics have also pointed out that Sarepta did not disclose the LGMD patient’s death during a recent corporate update, increasing investor and patient concerns.
