FDA Leadership Shakeup: Nicole Verdun's Exit Marks Significant Shift in Cell and Gene Therapy Regulation

In a move that has sent shockwaves through the pharmaceutical industry, Nicole Verdun, the director of the FDA's Office of Therapeutic Products (OTP), was placed on administrative leave alongside her deputy, Rachael Anatol. This unexpected development comes amid a series of changes at the agency, signaling a potential shift in the regulation of cell and gene therapies.

Verdun's Departure and Its Implications

Nicole Verdun, who had been with the FDA since 2012, was appointed director of the OTP in 2023. Her role was crucial in managing the increasing number of cell and gene therapy (CGT) products requiring FDA attention. Verdun's exit has left industry professionals stunned, as she was widely regarded as a stabilizing force following the forced resignation of Peter Marks, the former head of the Center for Biologics Evaluation and Research (CBER).

The timing of Verdun's departure is particularly significant, coming shortly after the controversial approval of Sarepta's gene therapy, Elevidys, for Duchenne muscular dystrophy. Verdun had reportedly disagreed with Marks on this approval, which has recently been linked to the deaths of two teenage patients.

Power Consolidation Under Vinay Prasad

Concurrent with Verdun's exit, Vinay Prasad, the current CBER Director, was named the FDA's chief medical and scientific officer – a role traditionally split between two individuals. This move has been interpreted by some as a consolidation of power within the agency.

Prasad, formerly a professor at the University of California, San Francisco, has been a vocal critic of certain FDA decisions, including the approval of Elevidys. His expanded role and the removal of Verdun and Anatol suggest a potential shift in the FDA's approach to cell and gene therapy regulation.

Industry Reactions and Concerns

The pharmaceutical industry has expressed deep concern over these developments. Emil Kakkis, CEO of Ultragenyx, stated that Verdun's loss "represents a further degradation in the base of experienced and knowledgeable leadership that will not readily be replaced."

The Alliance for Regenerative Medicine also voiced disappointment, praising Verdun and Anatol for modernizing the FDA's regulatory approach to CGT and maintaining the agency's global leadership in this evolving field.

These changes at the FDA come at a critical time for the cell and gene therapy sector, raising questions about the future direction of regulation and approval processes for these innovative treatments.