CAMBRIDGE, Mass. — Sarepta Therapeutics (NASDAQ: SRPT) announced Tuesday that it will voluntarily and temporarily pause all shipments of its Duchenne muscular dystrophy gene therapy, ELEVIDYS (delandistrogene moxeparvovec), in the United States, effective at the close of business on July 22.
The decision, according to the company, is a proactive measure intended to give Sarepta and the U.S. Food and Drug Administration (FDA) time to complete the safety labeling supplement process for the therapy and to address any outstanding regulatory questions.
“As a patient-centric organization, the decision to voluntarily and temporarily pause shipments of ELEVIDYS was a painful one, as individuals with Duchenne are losing muscle daily and in need of disease-modifying options,” said Doug Ingram, Sarepta’s chief executive officer. “It is important for the patients we serve that Sarepta maintains a productive and positive working relationship with FDA, and it became obvious that maintaining that productive working relationship required this temporary suspension.”
Sarepta emphasized that the pause is not due to any newly identified safety concerns, and the company remains committed to working closely with the FDA through a collaborative and science-based review process.
ELEVIDYS is a one-time, adeno-associated virus (AAV)-based gene therapy designed to address the root cause of Duchenne muscular dystrophy (DMD) — mutations in the DMD gene that prevent the production of dystrophin, a key structural protein in muscle cells. The therapy delivers a transgene that encodes ELEVIDYS micro-dystrophin, a shortened but functional version of the protein.
The therapy is currently approved for intravenous use in DMD patients aged four and older who have a confirmed DMD gene mutation. It is indicated for both ambulatory and non-ambulatory patients. The non-ambulatory indication is authorized under the FDA’s accelerated approval pathway, contingent on confirmatory trials demonstrating clinical benefit.
Sarepta pledged to provide timely updates to patients, healthcare providers, and the wider Duchenne community as more information becomes available.
