FDA Taps Biotech Exec for RFK Jr.’s Drug Regulator Role
FDA’s top Drug Reviewer Steps Down Amid Scrutiny Over Controversial Approvals
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Dr. Peter Marks, a pivotal figure in the Food and Drug Management’s drug approval process, is departing his role as director of the Center for Drug Evaluation and Research (CDER). His tenure has been marked by important decisions,including the controversial accelerated approval of Biogen’s Alzheimer’s drug,Aduhelm,and the expansion of Sarepta Therapeutics’ Duchenne muscular dystrophy treatment,Elevidys.
A Shift in Leadership at a Critical juncture
The departure of Dr. marks comes at a time of intense focus on the FDA’s regulatory practices, especially concerning novel therapies and accelerated approval pathways.His leadership at CDER has overseen the review of numerous groundbreaking treatments, but also faced considerable debate and criticism.
Marks’s decisions have often placed him at the center of high-stakes debates within the pharmaceutical and medical communities. The approval of Aduhelm, for instance, was met with widespread skepticism due to questions surrounding its efficacy and the FDA’s reliance on a controversial advisory committee vote. Similarly, the expanded approval of sarepta Therapeutics’ elevidys for Duchenne muscular dystrophy has drawn attention, especially in light of recent safety concerns.
Last week, the FDA requested that Sarepta Therapeutics halt all shipments of Elevidys following the deaths of three patients from liver failure after receiving the treatment or a similar one. Sarepta afterward stated it would not cease shipments for patients with the condition who can still walk, asserting that data indicated “no new or changed safety signals” within this patient group.
In an interview with CNBC prior to the proclamation of his successor, Marks commented on his past decisions regarding gene therapy, stating they were “made on the best available knowledge at the time.” He noted that the primary debate at that juncture centered on efficacy rather than safety.
Marks expressed that he doesn’t consider it “unreasonable” to ask Sarepta to pause shipments pending a thorough review of all relevant information.
Tidmarsh Tapped to Lead CDER
Stepping into the role of CDER director is Dr. Nicole Tidmarsh, a physician-scientist with extensive experience in drug progress. Tidmarsh’s background includes leadership positions at several prominent biotech firms, including serving as CEO of La Jolla pharmaceuticals and Horizon Pharma, which she founded before its acquisition by Amgen for $28 billion. She also founded Threshold Pharmaceuticals and held senior roles at other biotechnology companies.
“Dr. Tidmarsh is an accomplished physician-scientist and leader whose experience spans the full arc of drug development-from bench to bedside,” said FDA commissioner Dr.Marty Makary in a statement. “Her appointment to lead CDER brings remarkable scientific, regulatory, and operational expertise to the agency.”
Tidmarsh’s leadership is expected to influence the FDA’s approach to the accelerated approval process and its oversight of prescription drug advertising, areas that have been under particular scrutiny during Marks’s tenure.
— CNBC’s Angelica Peebles contributed to this report