Sarepta to Pause Shipments of the Gene Therapy Elevidys

Sarepta Therapeutics will temporarily stop shipping Elevidys (delandistrogene moxeparvovec) in the United States at the end of business today, July 22, 2025. This reverses its decision from a few days ago to keep selling the gene therapy, which treats patients with Duchenne muscular dystrophy.

Company officials said the temporary halt in sales will allow Sarepta and the FDA to complete the safety labeling update for Elevidys.

Doug Ingram

”It is important for the patients we serve that Sarepta maintain a productive and positive working relationship with the FDA, and it became obvious that maintaining that productive working relationship required this temporary suspension while we address any questions that the FDA may have and complete the Elevidys label supplement process,” Doug Ingram, CEO of Sarepta, said in a news release.

Related: FDA Wants Elevidys Off the Market. Sarepta Said No.

The FDA on Friday had requested that Sarepta stop selling the gene therapy and placed on hold the clinical trial of Elevidys to treat limb-girdle muscular dystrophy following three patient deaths, saying the patients are or would be exposed to an unreasonable and significant risk of illness or injury.

At that time, Sarepta had said there was no need to stop offering Elevidys to ambulatory patients. Earlier this month, the company had stopped shipping the therapy for non-ambulatory patients with Duchenne muscular dystrophy after a second patient died from acute liver failure. The company has also paused a phase 3 study that is a confirmatory trial for the gene therapy’s use with non-ambulatory patients.

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. 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.

Elevidys is approved for patients aged 4 and older with a confirmed mutation in the DMD gene, both those who can walk and those who cannot. For non-ambulatory patients, the approval is accelerated and requires a confirmatory trial.

FDA officials said they had made the requests for market removal because of concern about Sarepta’s AAVrh74 platform technology used in Elevidys and in another investigational gene therapy in a phase 1 trial where a patient also died. Regulators have revoked approval for the AAVrh74 platform technology, saying there isn’t sufficient evidence to demonstrate the technology’s safety.

Sarepta said that investigational gene therapy, SRP-9004, was manufactured with a different process. In an investor call, Ingram 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.