STATEMENT RELEASE: Doctors For America’s FDA Task Force’s Statement on the FDA’s Decision to Ignore Patient Safety in Recent Decision on Sarepta’s Gene Therapy for the Treatment of Duchenne Muscular Dystrophy

Doctors for America
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Washington, DC  20009-9997

June 22, 2023

Doctors For America’s FDA Task Force’s Statement on the FDA’s Decision to Ignore Patient Safety in Recent Decision on Sarepta’s Gene Therapy for the Treatment of Duchenne Muscular Dystrophy

WASHINGTON, D.C. – The FDA Task Force is committed to increasing transparency at the FDA and ensuring that improving patient outcomes including safety is at the heart of all therapeutic approvals. The task force has been closely following the consideration of Sarepta’s gene therapy, Elevidys (SRP-9001 or delandistrogene moxeparvovec), for the treatment of Duchenne muscular dystrophy (DMD).

As physicians, our priority is our patients’ health and wellness. We want patients, especially those with a rare and progressively fatal disease affecting 1 out of every 3,300 boys globally, to have hope for a treatment that works and that it is safe. Gene therapies offer incredible promise, but there must be careful consideration of these novel treatments especially when being considered for approval for use in young people, where safety concerns may manifest after the clinical trial period. 

Our physician lead, Dr. Reshma Ramachandran recently co-authored an op-ed on why FDA’s decision to grant Sarepta accelerated approval should be held for consideration of the therapy’s confirmatory trial. The FDA Task Force stands by the sentiments in this article, “The standard for accelerated approval does not appear to have been met here — to the contrary, the drug’s risks may outweigh its benefits. DMD patients need better treatment options, but they also need the FDA to uphold strong evidentiary standards so they can be confident in approved drugs.

Disappointingly, the FDA has approved Elevidys under the accelerated approval pathway, abandoning such high evidentiary standards and shifting the burden of uncertainty of the drug’s efficacy and safety onto patients and their doctors. Although the FDA has approved this gene therapy for patients aged 4-5 years old, the agency’s medical reviewers had themselves stated that the submitted clinical trial evidence “must be interpreted with caution” as the studies had not been designed to draw any conclusions of the treatment’s effect on this group. This narrowed indication also does not preclude off-label prescribing of Elevidys to other patients of other age groups, subjecting patients looking for hope to an unproven and expensive treatment.

Moreover, besides safety, there may be other significant tradeoffs with these treatments. Due to the immune response of patients undergoing gene therapy, they could be ineligible to receive another future and potentially more effective alternative. The FDA should have instead not approved this drug under accelerated approval and awaited the results of the Phase 3 clinical trial that is fully enrolled and ongoing, which would provide more definitive answers that the gene therapy indeed works and is safe. The decision to grant accelerated access to a costly therapy based on an unvalidated surrogate endpoint without clear association with meaningful clinical benefit lowers the bar on innovation and may waste precious time if companies follow suit and develop similar therapies of uncertain benefit. 

For press inquiries for members of the FDA Task Force Doctors for America please contact Claire Onyechi with Continuum Health Group ([email protected]).


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 and on Twitter drsforamerica.