Sarepta to Resume Shipping Elevidys For Ambulatory Duchenne Patients

The FDA notified Sarepta Therapeutics that it may lift its voluntary pause on shipments of Elevidys (delandistrogene moxeparvovec) for ambulatory patients with Duchenne muscular dystrophy. Sarepta will resume shipping the gene therapy to sites of care, the company announced in a news release.

The FDA’s review of the safety data in the ambulatory population included the case of an 8-year-old in Brazil whose death was deemed unlikely to be related to treatment with Elevidys by the Brazilian health authorities. FDA officials have concluded the death was unrelated to treatment.

Roche markets Elevidys outside the United States. Last week, regulators with the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) issued a negative opinion on the conditional marketing authorization in the EU.

Last week, the FDA had asked Sarepta to stop shipping Elevidys. After several days of negotiation with regulators, Sarepta agreed. Earlier this month, the company had stopped shipping the therapy for non-ambulatory patients with Duchenne 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.

Doug Ingram

In the news release, Sarepta CEO Doug Ingram said they continue to work with regulators to complete the safety label update for Elevidys, and they are discussing an approach for risk mitigation for 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.