Sarepta Therapeutics has announced a voluntary and temporary pause on all U.S. shipments of its Duchenne muscular dystrophy gene therapy, ELEVIDYS (delandistrogene moxeparvovec). This decision, effective July 22, 2025, was made to allow the company sufficient time to address any requests for information from the U.S. Food and Drug Administration (FDA) and to complete the necessary safety labeling supplement process for ELEVIDYS.

According to Sarepta's CEO, Doug Ingram, while the pause is a "painful one" due to the daily muscle loss experienced by Duchenne patients, it's crucial for Sarepta to maintain a "productive and positive working relationship with FDA." This temporary suspension is intended to facilitate a collaborative and science-driven review process and dialogue with the regulatory agency.

ELEVIDYS is a single-dose, adeno-associated virus (AAV)-based gene transfer therapy. Its mechanism of action involves delivering a transgene that codes for the production of ELEVIDYS micro-dystrophin in skeletal muscle, aiming to address the underlying genetic cause of Duchenne. The therapy is indicated for individuals at least four years of age with Duchenne muscular dystrophy, encompassing both ambulatory and non-ambulatory patients with confirmed DMD gene mutations. The indication for non-ambulatory patients was granted under accelerated approval, contingent on verification of clinical benefit in future confirmatory trials.

The company has also provided important safety information regarding ELEVIDYS. The therapy is contraindicated in patients with specific deletions in exon 8 and/or exon 9 of the DMD gene. The most common adverse reactions identified in clinical studies include vomiting, nausea, liver injury, fever, and thrombocytopenia. Sarepta assures continued transparency and will provide timely updates to the Duchenne community.