US FDA’s Top Cell and Gene Therapy Regulators Forced Out

Office of Therapeutic Products Director Nicole Verdun and her deputy Rachael Anatol were escorted out of FDA headquarters on June 18. Disagreements over CAR-T regulation and Capricor’s DMD treatment may be to blame.

Nicole Verdun at 2023 ASGCT Policy Summit
Nicole Verdun (left) speaking at a 2023 event shortly after becoming OTP director. • Source: Screenshot of conference livestream
Key Takeaways
  • OTP Director Nicole Verdun and Deputy Director Rachael Anatol have been forced out of the FDA.
  • A source familiar with the matter believed Verdun and Prasad disagreed over CAR-T regulation.
  • Industry officials recently praised Verdun’s leadership during industry conferences.

Top cell and gene therapy regulators Nicole Verdun and her deputy Rachael Anatol were forced out of the US Food and Drug Administration June 18, per multiple sources familiar with the matter.

A comment sent to Pink Sheet from Health and Human Services Department press secretary Andrew Nixon indicated Verdun and Anatol disagreed with Center for Biologics Evaluation and Research Director Viany Prasad.

Verdun and Anatol were director and deputy director of CBER’s Office of Therapeutic Products.

“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,” Nixon said.

Verdun and Anatol’s departures come along with other key CBER cell and gene therapy exits earlier this year and as CBER continues adapting to Prasad. He replaced Peter Marks, who was also forced out of the FDA by the Trump Administration.

Among those who left OTP in March were Lola Fashoyin-Aje, director of the Office of Clinical Evaluation, Heather Lombardi, director of the Office of Cellular Therapy and Human Tissue CMC, Heather Erdman, associate director of quality assurance in the Office of Review Management and Regulatory Review, and Vaishali Popat, branch chief of General Medicine Branch 3 in the Office of Clinical Evaluation.

One source familiar with the matter believed that Prasad and Verdun disagreed over the regulation of CAR-T products, as well as Capricor Therapeutics’ Duchenne muscular dystrophy drug deramiocel (CAP-1002). The FDA announced a July 30 advisory committee meeting for deramicoel, but then the agency abruptly pulled the meeting’s Federal Register notice.

The product has an Aug. 31 user fee goal date.

High Praise For Verdun From Industry

Several industry officials strongly praised Verdun and Anatol during a June 5 cell and gene therapy roundtable. Verdun was notably absent from the panel discussion despite being listed as co-moderator of the event one day prior.

The Pink Sheet also heard strong praise for her leadership at recent industry meetings, including the BIO annual convention this week and a National Organization for Rare Disorders meeting earlier this month.

Verdun and Anatol were hired to lead OTP, CBER’s only super office, in the past two years “and they became the reformers, that really said, ‘we have to change things,’” Timothy Hunt, CEO of the Alliance for Regenerative Medicine, said at FDA’s June 5 roundtable.

“Along with the loss of other senior FDA staff, this is a significant setback for cell gene therapy,” former FDA Commissioner Scott Gottlieb, who served during Trump’s first term, posted on X. “The advent of these advances was one of President Trump’s first term triumphs, along with his efforts to expand access to them. The field now faces a harder future.”

“These FDA leaders, with their deep subject matter expertise, knew how to make the pathway for these cell and gene therapies more viable,” Gottlieb wrote in another tweet. “They have been doing hard work to pave the way for safe and effective advances to reach patients who could benefit from them.”

At an early June NORD meeting Verdun highlighted her commitment to evolved thinking and urged companies with stalled cell and gene therapy programs to return to the FDA to see if the programs could be revived.

Prasad’s Past CAR-T Comments

Prasad previously has taken issue with CBER’s regulation of CAR-T therapies for multiple myeloma.

In a comment published online in Nature Reviews Clinical Oncology in May 2024, he criticized the Oncologic Drugs Advisory Committee’s endorsement of two CAR-T products, Janssen Biotech’s Carvykti (ciltacabtagene autoleucel) and Bristol Myers Squibb’s Abecma (idecabtagene vicleucel), for earlier treatment lines in multiple myeloma.

In contrast with acute lymphoblastic leukemia and large B cell lymphoma, CAR-T cells are not a curative treatment for relapsed/refractory multiple myeloma. The therapies have failed to demonstrate convincing survival improvements in such patients, Prasad said.

The use of surrogate endpoints lacking a robust correlation with overall survival, along with suboptimal control arms and use of post-progression therapies, limited the ability to make definitive conclusions on the basis of the available data, he said.

Prasad also cited the “formidable price tag” of both drugs, $370,000-$475,000, and their toxicity profiles.

Sue Sutter contributed to this report.

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