The well-respected director of the FDA’s cell and gene therapy office was seen as a stabilizing and trustworthy voice inside the quickly reshaping FDA, especially since the late-March exit of CBER Director Peter Marks.
Nicole Verdun, the influential director of the FDA’s Office of Therapeutic Products, was placed on administrative leave Wednesday afternoon. Her deputy, Rachael Anatol, was also put on leave.
Verdun had been with the agency since 2012 and was appointed director of the Office of Therapeutic Products (OTP), the agency’s cell and gene therapy division, in 2023 when it was established to deal with the deluge of cell and gene therapy (CGT) products that needed attention. Her departure has shocked industry professionals, who saw Verdun as a sign of stability following the forced resignation of her close colleague, former Center for Biologics Evaluation and Research head Peter Marks.
After Marks was pushed out in late March around the time Marty Makary was confirmed as FDA commissioner, “everyone exhaled and had a sigh of relief because Verdun remained, even because she disagreed with Marks occasionally,” Courtney Rice, a principal at the rare disease and gene therapy consultancy Acadia Strategy Partners, told BioSpace.
Continuity and institutional knowledge at the FDA are important in the CGT space, Rice added. “Every [CGT] product is unique. These aren’t one hits; these aren’t statins.”
Verdun butted heads with Marks on occasion, notably disagreeing with her boss as Sarepta’s gene therapy for Duchenne muscular dystrophy, Elevidys, moved toward approval. Marks ultimately greenlit the therapy, which has recently been linked with the death of two teenage patients with DMD, despite the dissent of the FDA advisory committee.
Current CBER Director Vinay Prasad, then a professor at the University of California, San Francisco, also disagreed with the decision, blasting Marks in March after a patient taking Elevidys died of liver failure caused by the treatment.
“It is rare that you can blame a single person for a regulatory failure, but Peter Marks at FDA overruled 3 review teams and pushed this product on the market despite a failed trial,” Prasad wrote on X.
“Nicole tilts more in line with Prasad, who’d been extremely vocal over his disagreement over Sarepta approval as a spectator,” Rice said. “I thought she was viewed as an ally of the current administration, or certainly not a threat.” Indeed, Verdun had survived the first sweep of changes coming over the FDA and CBER.
Her exit was widely regretted by the industry.
“Given that Dr. Verdun was a critically important part of the CBER organization in OTP, her loss represents a further degradation in the base of experienced and knowledgeable leadership that will not readily be replaced,” Ultragenyx CEO Emil Kakkis told BioSpace by email.
“Dr. Makary and Dr. Prasad have been on a listening tour for CEOs and so I would recommend they listen now and explain thoroughly why they are making this decision at this exact time in the context of so much instability and uncertainty at FDA,” Kakkis added.
The Alliance for Regenerative Medicine echoed Kakkis’ sentiment. “Over the past two years, Nicole and Rachel[sic] have modernized the FDA’s regulatory approach to cell and gene therapy (CGT), earning the trust and respect of the CGT community and helping to ensure that the FDA was the global leader in this evolving field,” ARM wrote in a statement Thursday. “We are deeply disappointed.”
Why Verdun and Anatol were placed on leave is still a bit of a mystery, but the move has the appearance of a consolidation of power, according to some observers.
“I think this was a consolidation of power, a power grab. They’re derisking by moving her aside.
They want to control the interaction with industry, every word of the interaction,” Rice said, “and the way you do that is to consolidate everything through one person and that’s Vinay Prasad.”
The same day that Verdun and Anatol were let go, Prasad was named the FDA’s chief medical and scientific officer, traditionally two roles held by two separate people.
According to STAT News, Prasad is not heavily involved in administrative tasks.
“Multiple agency [FDA] employees, who requested anonymity to protect against retaliation, told STAT that Prasad seemed uninterested in the administrative work of being a center director,” Don Fink, a former FDA employee, wrote in a LinkedIn post on Thursday. “One CBER agency official said Prasad rarely attends center meetings with office directors, and that their questions about the budget, HR functions, and IT systems have gone unanswered,” the post continued.
Rice has questions of her own. “There’s more to the story, I don’t know what it is,” she told BioSpace. “Escorting her out after 13 years? Well respected in an elevated office? It’s gratuitous. I think there’s some emotion, I think there’s more to this story. Time will tell.”
