FDA Wants Elevidys Off the Market. Sarepta Said No.

The FDA has asked Sarepta Therapeutics to stop shipping Elevidys (delandistrogene moxeparvovec), its gene therapy for Duchenne muscular dystrophy, but according to regulatory officials, Sarepta leaders refused.

“Based on our comprehensive scientific interpretation of the data, which shows no new or changed safety signals in the ambulant patient population, we will continue to ship Elevidys to the ambulant population,” Sarepta said in a statement. “We look forward to continued discussions and sharing of information with the FDA in order to advance our shared purpose of protecting patient safety and informed access to care.

The FDA has also placed on hold the clinical trial of Elevidys to treat limb girdle muscular dystrophy following three deaths patients, saying in a news release late on Friday that the patients are or would be exposed to an unreasonable and significant risk of illness or injury.

FDA officials said the three deaths appear to have been a result of acute liver failure in individuals treated with Elevidys or investigational gene therapy using the same AAVrh74 serotype that is used in Elevidys. One of the fatalities occurred during a clinical trial conducted under an investigational new drug application for the treatment of limb girdle muscular dystrophy.

In the statement, Sarepta officials said the 51-year-old non-ambulant limb-girdle muscular dystrophy (LGMD) patient who died was participating in a phase 1 trial. The patient was treated with a different dose of a gene therapy, SRP-9004, which was manufactured with a different process. The patient had not been treated with Elevidys, Sarepta officials said.

Doug Ingram

In a call with investors before the FDA’s announcement, Doug Ingram, president and CEO of Sarepta, said the company had decided not to proceed with further development of SRP-9004. That decision, however, was made not based on the safety of the therapy or issues related to acute liver failure. The company is prioritizing instead its siRNA platform to support its long-term financial viability

He said the company will present the limb-girdle results at a medical meeting when the full data are available.

Related: Sarepta Stops Shipments of Elevidys For Non-Ambulatory DMD Patients

Sarepta officials said they stopped shipments of Elevidys for non-ambulant patients while they work with the FDA to update the label and evaluate the use of an enhanced immunosuppression regimen to mitigate the risk of acute liver failure.

Last month, the company said it paused a phase 3 study that is a confirmatory trial for Elevidys for use with non-ambulatory patients. The company has put together an independent group of experts in Duchenne and liver health to consider an enhanced immunosuppression regimen for Elevidys. The panel will evaluate the data for adding the immunosuppression drug sirolimus to patients with Duchenne who are non-ambulatory.

Elevidys received traditional approval for use in ambulatory Duchenne muscular dystrophy patients 4 years of age and older on June 20, 2024. It was approved for non-ambulatory patients on June 22, 2023, under the accelerated approval pathway.

Elevidys is a one-time, intravenous treatment that uses adeno-associated virus (AAV) technology to deliver a gene designed to produce a shortened form of the dystrophin protein, known as Elevidys micro-dystrophin, directly to skeletal muscle.

Regulators also said they’ve revoked approval for Sarepta’s AAVrh74 Platform Technology used in Elevidys, saying there isn’t sufficient evidence to demonstrate the technology’s safety.

Sarepta officials The FDA has requested that the label include a black box warning for acute liver injury (ALI) and acute liver failure (ALF). Sarepta agrees with this change, which they said appears to resolve any material issues with the ambulant portion of the Elevidys label.

Elevidys is priced at $3.2 million per dose. Sarepta had $1.7 billion in net product revenue in 2024, almost half of which — $821 million — came from Elevidys. Ingram said during today’s investor call the company will reassess revenue guidance and the impact on the second quarter.