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]).
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About Doctors for America:
Doctors for America mobilizes doctors and medical students to be leaders in putting patients over politics to improve the health of our patients, communities, and nation. DFA is an organization of over 27,000 physician and medical student advocates in all 50 states, representing all areas of specialization. DFA teaches physicians and medical students advocacy skills and does advocacy at a state and federal level. Our impact areas focus on access to affordable care, community health and prevention, and health justice and equity. DFA focuses solely on what is best for our patients, not on the business side of medicine, and does not accept any funding from pharmaceutical or medical device companies, insurance companies or for-profit healthcare companies; which uniquely positions DFA as the organization that puts patients over politics and patients over profits. Find out more at doctorsforamerica.org and on Twitter drsforamerica or Bluesky drsforamerica.bsky.social.