CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Sarepta Therapeutics, Inc. (SRPT) , the leader in precision genetic medicine for rare diseases, today issued the following statement:
Shortly after 2:30 p.m. ET today, Sarepta received an informal request from the U.S. Food and Drug Administration (FDA) to voluntarily halt shipment of ELEVIDYS (delandistrogene moxeparvovec), our gene therapy for Duchenne muscular dystrophy (Duchenne), in the U.S. We first heard of this potential request earlier in the day at the same time the public and our patient communities did, through media reports.
At Sarepta, patient safety and well-being are always our top priority. We are committed to upholding the highest safety standards for all of our therapies. This guides every decision we make, as evidenced by our conservative decision to pause shipments of ELEVIDYS for non-ambulant patients while we work with the FDA to update the label and evaluate the use of an enhanced immunosuppression regimen to mitigate the risk of acute liver failure.
Based on our comprehensive scientific interpretation of the data, which shows no new or changed safety signals in the ambulant patient population, we will continue to ship ELEVIDYS to the ambulant population. We look forward to continued discussions and sharing of information with FDA in order to advance our shared purpose of protecting patient safety and informed access to care.
We recognize that the death of any patient is heartbreaking, including the recent death of a 51-year-old non-ambulant Limb-Girdle Muscular Dystrophy (LGMD) patient. We also want to clarify that this tragic event occurred in a Phase 1 clinical trial for an investigational gene therapy called SRP-9004. SRP-9004 is a clinical stage therapy that is intended to treat a different disease (LGMD Type 2D), is administered using a different dose, and is manufactured using a different process. The LGMD study participant who passed away was not treated with ELEVIDYS, and the dosing for the SRP-9004 trial had concluded at the time of his death.
Additionally, in a timely manner, Sarepta reported this ALF event as a life-threatening case to FDA on June 20, 2025, and further followed up with notification to FDA of the death on July 3, 2025, in accordance with applicable law and our commitment to full regulatory transparency.
ELEVIDYS is the only approved gene therapy for individuals devastated by Duchenne, a rare, progressive and ultimately fatal disease. We are committed to working closely with the FDA to ensure that all decisions are grounded in science and the best interests of patients, considering the compelling need of these families to access disease-modifying therapy. |
