FOR IMMEDIATE RELEASE
Doctors for America
PO Box: 21161
2300 18th St NW Lbby
Washington, DC 20009-9996
June 24, 2024
Doctors for America’s FDA Task Force Strongly Condemns FDA Approval of Elevidys
Washington, D.C.: Last week, the U.S. Food and Drug Administration (FDA) granted Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy, Elevidys, full approval for use in patients four years and older. Elevidys had been previously approved through the accelerated approval pathway for the treatment of Duchenne’s muscular dystrophy in patients four to five years old. In the confirmatory trial required under accelerated approval, Elevidys failed the primary clinical endpoint. This stunning decision despite the negative findings and objections from multiple FDA review teams undermines standard FDA operating procedures, disregards scientific data, and endangers the agency’s credibility and public trust.
Doctors for America’s FDA Task Force strongly condemns the FDA’s approval of Elevidys, which was approved despite its failure to confirm clinical benefit. As physicians, we’ve witnessed first-hand the devastating impact Duchenne muscular dystrophy has on patients and families, and we understand the importance of expanding treatment options for this rare disease. However, the approval of an extremely expensive but effectively uncertain drug is ultimately a disservice to patients.
Further, we are deeply alarmed that this approval appears to have been entirely the decision of one individual, FDA’s Center for Biologics Evaluation and Research (CBER) Director Peter Marks. Three separate review teams expressed their objections to the initial accelerated approval and then again to this conversion to full and expanded approval. However, in both instances, the CBER Director overruled expert agency staff and proceeded ahead with approval, including clinical trial evidence that clearly fails to demonstrate clinical benefit. The Center Director also seems to have ignored warnings from his own staff on the undue impacts of this approval in that 1) patients in taking this unproven gene therapy will likely be unable to receive another more promising gene therapy due to safety concerns; and 2) full approval of Elevidys will make “infeasible” further collection of robust data to determine whether there are any subgroups of patients who could benefit from this drug.
These instances of approvals based on subjective notions rather than scientific evidence demonstrate the need for an external evaluation of the FDA’s procedures and regulations, and consideration of potential reforms to agency policies. The public should have confidence that no single FDA official has the ability to overrule multiple expert review teams and top lieutenants to unilaterally approve a drug.
Physicians and patients rely on the FDA to serve as the ‘gold standard’ for drug approvals. We need to be able to trust that any drug that the FDA approves has been fully proven to be safe and effective. All patients, including those suffering from a devastating, progressive disease such as Duchenne’s muscular dystrophy deserve a regulator that ensures that approved treatments are both safe and effective. As DFA’s FDA Task Force, we are deeply disheartened by last week’s decision and urge the FDA to reconsider the full approval of Elevidys and require Sarepta Therapeutics to conduct additional confirmatory studies.
For press inquiries and to speak more on this issue with members of the Doctors for America FDA Task Force, please contact Claire Onyechi 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, 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.