Sarepta Pauses DMD Gene Therapy Shipments Following FDA Request

July 24, 2025 – Sarepta Therapeutics has implemented a voluntary, temporary pause on all U.S. shipments of its Duchenne muscular dystrophy (DMD) gene therapy, Elevidys, effective July 22. This decision follows an FDA request made on July 17 and aims to preserve a “productive working relationship” with the agency.

The move comes during a challenging period for Sarepta, marked by multiple patient deaths and increased regulatory scrutiny. A third patient death in an LGMD gene therapy trial (SRP-9004), coupled with two prior Elevidys-linked deaths from acute liver failure (ALF), prompted FDA action. The agency also placed clinical holds on SRP-9004 trials and revoked Sarepta’s AAVrh74 platform designation.

Sarepta is working with the FDA to address questions and update Elevidys’ safety label, which now includes a black box warning for acute liver injury (ALI) and ALF. The company also announced 500 job cuts as part of a restructuring. The future for Duchenne patients and Sarepta’s therapies remains uncertain.