Sarepta Therapeutics Temporarily Stops ELEVIDYS Shipments to Address FDA Request

Sarepta Therapeutics Pauses ELEVIDYS Shipments in the US to Play a Vital Role

Announcement

Sarepta Therapeutics, Inc., a known producer of precision genetic medicine for rare diseases, decided to temporarily and voluntarily halt all shipments in the United States for Elevidys (delandistrogene moxeparvovec) for Duchenne muscular dystrophy, effective from 22nd July 2025. Sarepta informed the US Food and Drug Administration (FDA) regarding the pause. This decision will give Sarepta the required time to respond to the request regarding information and will also let FDA and Sarepta look after the completion of ELEVIDYS safety labelling supplement work. Sarepta is consistence towards its commitment to patient safety and transparency and ensures to reach the timely updates to healthcare providers, broader Duchenne community, and patients as soon as the updated information is available. The decision will enhance the presence and enable ELEVIDYS to better serve the patients.

Details of ELEVIDYS

ELEVIDYS is a single dose for adeno-associated virus (AAV) powered gene transfer therapy for intravenous infusion introduced to identify the genetic cause of Duchenne muscular dystrophy, which is a change or mutation in the DMD gene resulting in reduced dystrophin protein. It happens via the delivery of a transgene that codes for the fixed production of ELEVIDYS micro-dystrophin in skeletal muscle. ELEVIDYS is a treatment of Duchenne muscular dystrophy (DMD) in the population of the 4-year age group. The trial discovered acute serious liver injury in ELEVIDYS individuals, and the administration might accelerate liver enzymes like ALT, GGT, AST, and GLDH or total bilirubin, generally visible within 8 weeks.

The immune-mediated myositis has also been identified around one month following ELEVIDYS infusion in individuals with deletion mutations engaging exon 8 and/or exon 9 in the DMD gene. Further, the safety information and warning/precautions have been detailed for better understanding at www.fda.gov/medwatch.

Views and Statements

The chief executive officer of Sarepta, Doug Ingram, said, “been a patient-centric company, the decision the pause shipments of Elevidys was heartbreaking for us as people with Duchenne are facing muscle loss regularly and are looking for disease-modifying options. It is essential for the patients we provide that Sarepta keep a positive and productive working relationship with the FDA, and it happens to be normal to do so, as it’s a requirement for both a temporary pause to identify the questions that only the FDA has. Also, along with it, we could complete the ELEVIDYS label supplement process.”