The Alliance for Regenerative Medicine has voiced deep disappointment about reports that the FDA has put the director of its cell and gene therapy office on administrative leave and escorted her out of the agency.
Stat reported Nicole Verdun, M.D., director of the FDA’s cell and gene therapy office, and her deputy Rachael Anatol, Ph.D., were relieved of their duties without being given an explanation. Pushing Verdun out of the FDA would intensify questions, which date back to at least the exit of Peter Marks, M.D., Ph.D., about the agency’s ability to provide a predictable service to cell and gene therapy developers.
In recent months, multiple biotechs have responded to analyst questions about changes at the FDA by praising Verdun and highlighting the continuity she provided. Leaders from Capricor Therapeutics, Lexeo Therapeutics, Intellia Therapeutics and Sarepta Therapeutics are among those to name-check Verdun on earnings calls and at investor events this year. Taysha Gene Therapies CEO Sean Nolan provided insights into Verdun’s importance last month.
“Verdun has certainly been at the official meetings. We've had ... consistent senior leadership review. Peter was never in any of the meetings, that's not his role. He leaves that up to Nicole and the teams beneath her. That part has been very consistent. There really has been no departure, no deviation from the conversations that we had going back to, call it, April of last year,” Nolan said on an earnings call.
A Department of Health and Human Services spokesperson commented on the development in an email to Fierce Biotech. “Center directors deserve to be supported by managers that are aligned with aggressive goals to expeditiously advance therapeutics for rare diseases using the gold standard of science,” the spokesperson said.
Biotechs have publicly praised Verdun’s role in accelerating the development of rare disease therapies. Talking on an earnings call last month, Sarepta CEO Doug Ingram said, “Verdun appears to be quite innovative in her goal of moving therapies along and relying upon modern tools for drug development.” Rocket Pharmaceuticals CEO Gaurav Shah, M.D., noted Marks and Verdun’s impacts in November.
“We have seen significant progress in flexibility around trial design, relying more on natural history, more openness to single-arm trials, and really being thoughtful about endpoints that don't take years, but take maybe one to two years,” Shah said at an investor event. “I think they recognize that part of safety is preventing fatal diseases.”
The comments suggest Verdun’s approach is aligned with views expressed by Vinay Prasad, M.D., who replaced Marks as head of the Center for Biologics Evaluation and Research earlier this year. Prasad has outlined plans for the FDA to use surrogate endpoints to make rare disease drugs available as soon as possible and then collect more information about safety and efficacy in the real world.
Analysts at William Blair said in a note to clients that: “Overall, while we view the departure of Drs. Verdun and Anatol as another sentiment hit and source of volatility for the cell and gene therapy spaces, we highlight that in recent weeks Drs. Prasad and Makary have continued to be outspoken in their support of cell and gene therapies, specifically those for the treatment of rare diseases.
“Therefore, we do not ultimately think these departures from the agency will significantly impact registrational trial designs and packages that have already been aligned on or will be announced soon.” The firm added, however, that it does “believe the FDA could require more stringent post-marketing safety monitoring and reporting and/or increase the requirements for patient disclosures and consent regarding data handling and safety.”