The latest shake-up at the Food and Drug Administration forced out the top two officials in charge of reviewing cell and gene therapies, analysts said Thursday and Friday.
Nicole Verdun, who directs the FDA's Office of Therapeutic Products, and her deputy, Rachael Anatol, were placed on administrative leave, William Blair analyst Sami Corwin said in a report. The OTP is under the Center for Biologics Evaluation and Research. Vinay Prasad recently took over as director at CBER after Peter Marks resigned amid a feud with new health secretary, Robert F. Kennedy Jr.
Verdun's exit adds uncertainty to the cell and gene therapy space, Leerink Partners analyst Mani Foroohar said in a note.
"While details remain sparse, we see this departure as consistent with Dr. Prasad's expanded role at FDA/CBER — this week expanded to FDA's Chief Scientific Officer and Chief Medical Officer," he said. "Dr. Verdun's departure will be taken as a clear negative to sentiment."
Gene therapy and editing stocks all slipped. Crispr Therapeutics and Beam Therapeutics closed down a respective 0.4% and 1.4%, while Sarepta Therapeutics stock fell 3.3%. In the cell therapy space, shares of Legend Biotech 2.6% and Arcellx stock fell 1.3%. Gilead Sciences shares, though, rose 0.5%.
A Shake-Up Under Vinay Prasad
The exit of Verdun and Anatol is just the latest in a series of high-profile departures from federal health agencies as the Trump administration takes hold.
Days after Kennedy was confirmed as health secretary, former CBER director Marks exited the agency. Marks cited disagreements with RFK Jr. over vaccine policy, accusing him of "misinformation and lies" in his resignation letter. Prasad, who has criticized some key gene therapy approvals, was tapped to lead CBER in May.
William Blair's Corwin says Prasad and new FDA Commissioner Marty 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," she said.
Why Sarepta Is At Risk
But the news could impact companies like Sarepta. Sarepta recently reported a second teenager died following treatment with its gene therapy, Elevidys. Elevidys treats Duchenne muscular dystrophy, a muscle-wasting disease that worsens over time.
Verdun reportedly sided with the review team, which initially suggested the Sarepta drug shouldn't be approved, Leerink's Foroohar said. Prasad, too, has criticized the approval. Marks, the former CBER director, overruled the team's decision, which is how Elevidys reached the market.
"It's unclear what this signals," Foroohar said. "Is Prasad simply cleaning house, or was there a deeper philosophical rift between him and Verdun over the agency's future direction?" he said. "We note that there is some discussion that Verdun and Prasad disagreed over the regulation of CAR-T products."
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