FOR IMMEDIATE RELEASE
Doctors for America
PO Box: 21161
2300 18th St NW Lbby
Washington, DC 20009-9996
October 11, 2024
Doctors for America Responds to Advisory Committee Vote on Barth Syndrome Therapy Elamipretide
Washington, DC: Doctors for America is deeply concerned that the FDA’s Cardiovascular and Renal Drugs Advisory Committee voted to recommend that the agency approve Stealth BioTherapeutics’ Barth syndrome therapy elamipretide in a 10-6 vote despite a clear lack of evidence from multiple studies supporting the therapy’s effectiveness.
As doctors, we understand the urgent need for therapies to treat our patients with ultra-rare diseases like Barth syndrome, but we need access to treatments that work and are proven to be safe. Doing so otherwise would give our patients false hope. While we support the continued use and opportunity for a panel of independent, expert advisors to review new drug application data, in this instance, we believe that this overall vote was based on stories, rather than science. In fact, the advisors acknowledged that their affirmative votes were cast in consideration of “anecdotal evidence,” which flies in the face of the valuable role that these advisory panels hold in critically evaluating scientific evidence to make an informed recommendation to the FDA. As FDA’s own reviewers point out
in their briefing document, the scientific evidence is clear. In its Phase II trial, the drug under consideration clearly fell short of its primary endpoints and failed to reach statistical significance for any secondary endpoints. Moreover, the drug certainly should not be approved based on the proposed surrogate biomarker, which is not correlated to any reliable clinical outcomes for the affected patients.
We call on the FDA to follow the science and protect our patients from false hope and potential harm, by not approving elamipretide for approval in their final review early next year. While regulatory flexibility may be appropriate for particular conditions, it should not be exercised when the scientific evidence as with elamipretide is clear in failing to demonstrate any meaningful efficacy for patients.
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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. We are 27,000 physicians and medical students in all 50 states, representing all areas of specialization. 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; which uniquely positions DFA as the organization that puts patients over politics and patients over profits. Find out more at doctorsforamerica.org and on X @drsforamerica.
