FDA Allows Sarepta to Resume Its AAV Gene Therapy Shipments for Ambulatory Duchenne Patients

In an unexpected turn, the Food and Drug Administration (FDA) has granted Sarepta Therapeutics permission to resume shipping its AAV gene therapy, Elevidys, to Duchenne muscular dystrophy (DMD) patients who can still walk. This decision comes just over a week after the agency had demanded a halt to all sales due to safety concerns.

Sarepta announced Monday evening that shipments to treatment sites would begin “imminently.” However, the voluntary pause on shipments for non-ambulatory Duchenne patients remains in effect. This pause was instituted by Sarepta in June, following the second death of a teenager from liver failure after receiving Elevidys. Both teenagers who died were non-ambulatory.

“We look forward to working collaboratively with the FDA to complete the safety label update for Elevidys and to discussing the approach to risk mitigation for nonambulatory patients, who remain on pause pending the outcome of those discussions,” stated Sarepta CEO Doug Ingram.

The FDA’s initial demand on July 18 to halt shipments to ambulatory patients had led to a three-day standoff with Sarepta, which initially refused to comply before backing down. This demand followed controversy over Sarepta’s failure to disclose a patient death in a clinical trial involving a different experimental gene therapy that shares a common AAV delivery component with Elevidys. More recently, an 8-year-old boy in Brazil who received Elevidys died, but his death was ruled unrelated to the gene therapy by the FDA.

The FDA’s sudden policy reversal caught Wall Street analysts by surprise. Previous reports, citing anonymous agency officials, suggested new safety data would be required. Vinay Prasad, the new head of the FDA’s gene therapy office, had also been a vocal critic of Elevidys prior to the patient deaths.

However, the FDA’s statement indicated it considered the “important voice” of the Duchenne patient community, a highly mobilized group with historical influence on agency decisions and, in some cases, funding ties to drugmakers like Sarepta. Analysts like Baird’s Brian Skorney suggest the “unexpected” reversal could stem from a “mix of patient advocacy pressure and a rebuke of FDA leadership by higher-ups” in the Trump administration, even hinting at potential resignations.

Elevidys’ renewed availability is expected to provide financial relief to Sarepta, which relies on the therapy for a minimum of $500 million in annual sales to remain viable. Analysts believe the decision increases the likelihood of Sarepta covering its debts. Despite this, some market observers, including William Blair’s Sami Corwin and RBC Capital Markets’ Brian Abrahams, anticipate “some hesitancy” from patients and physicians, predicting potential declines in Elevidys use below sales targets. Leerink Partners’ Joseph Schwartz warned that “even one death in an ambulatory patient could be the end of Elevidys.”