A Top FDA Official Overrules Reviewers’ Rejection of Sarepta’s DMD Gene Therapy


It looked as if the fate of Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy was pretty much sealed until an FDA official intervened. This information was provided by three people with knowledge of the FDA deliberations. The three staff members spoke on the condition of anonymity.

Prior to the FDA official’s intervention, FDA reviewers reached a non-binding conclusion that Sarepta’s gene therapy must be rejected. This prompted immediate intervention by Peter Marks, one of the top FDA officials and advocate for accelerated approvals of gene therapy. Peter Marks immediately requested that the staff schedule a public hearing on the gene therapy on May 12th, 2023.

Supporting the Stakeholder

Advocates as well as sponsors with an interest in shaping the FDA’s new mandate would like confirmation that firms will not be committed to investments in surrogate endpoints before they are validated. Surrogate endpoints are the benefit or even the likely benefit used as a measure of clinical outcome. They are used to determine whether the drug outweighs adverse effects such as drug-induced injury to the liver.

FDA’s Peter Marks Lends a Hand to Sarepta’s DMD Gene Therapy

 Although the FDA review teams did not recommend approving Elevidys, Peter Marks’ assessment was that data in a smaller group of patients between four and five years of age, justified accelerated approval. He agreed though that any expansion beyond this particular age-restricted group must depend on data from the EMBARK confirmatory trial.

Additional Ethical Support

The director of NYU Grossman’s medical school, Arthur Caplan, commented that even if Peter Marks moves against a vaccine candidate, his superiors could still override his decision.

The assistant secretary for HHS public affairs added that he has “never met” even one FDA regulator who would not resign his post if he received undue pressure in either direction. He continued that pressure of this sort undermines the confidence in our public health agencies.

For his part, Marks during a conference call last week with government officials, pharmaceutical executives, and academics who are part of a vaccine working group formed by NIH said he will resign if FDA approves a vaccine that hasn’t been proven to be safe and effective.

Peter Marks later re-emphasized these statements with Reuters. He added that he hasn’t felt any political pressure. However, he noted that if that changed, he “could not stand by and see something that was unsafe or ineffective being approved.”

He said that his resignation would be a signal to the American public that there was something wrong.

Update 6/30/2023: Sarepta’s $3.2 million Duchenne muscular dystrophy (DMD) gene therapy, Elevidys, won a long-awaited accelerated approval Friday in a restricted patient group. Data from an ongoing trial will follow.

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia (AML) six years ago. During this period of partial remission, Rose researched investigational drugs to be prepared in the event of a relapse. Her husband died February 12, 2021 with a rare and unexplained occurrence of liver cancer possibly unrelated to AML.

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