Statement on Elevidys Removal and Subsequent Return to Market

FOR IMMEDIATE RELEASE Doctors for America PO Box: 21161 2300 18th St NW Lbby Washington, DC 20009-9996 July 30, 2025 Doctors for America Statement on Elevidys Removal and Subsequent Return to the Market WASHINGTON, D.C. – Doctors for America’s FDA Task Force was pleased to see the FDA’s decision to suspend the distribution of Elevidys in the U.S. market after the third death of a patient who received a different but related gene therapy. Our condolences go out to the families who lost loved ones on this therapy and to those who received false hope that this therapy could be a viable treatment for a devastating disease. FDA’s decision to remove the voluntary hold for ambulatory patients who receive Elevidys while keeping the hold for non-ambulatory patients raises serious concern. Doctors for America strongly opposed the FDA’s decision to approve this therapy last year. It was clear then as it is now that the data did not prove efficacy or safety. The FDA acted in the interest of our patients by requesting a halt to U.S. shipments of this therapy. The ability of Sarepta to initially refuse to halt distribution demonstrates the limitations of the current system of voluntary withdrawal of a flawed product. The approval of medical products must be transparent and based on sound medical reasoning. Elevidys could not meet primary endpoints in clinical studies and it is well known that patients who are immunosuppressed, as muscular dystrophy patients are considered to be when treated with steroids, are at risk for serious liver toxicity with adenovirus vector drugs. The FDA must have the oversight power and regulatory authority in the post-approval real world setting to protect Americans from products that cause undue harm. The step of requesting distribution halt is justified when lives may be at risk, however a clear explanation that states the FDA’s reasoning in support of this unusual step is needed. Both patients and prescribing doctors deserve the full facts about this drug, and the assurance that expedited withdrawal of accelerated approval will follow distribution halt to protect patients from harm. For press inquiries and to speak more on this issue with members of Doctors for America, please contact Alli Everton with Continuum Health Group ([email protected]). ### About Doctors for America: