On June 20 of last year, the FDA handed Vijay Kumar one of the most consequential desks in American medicine, and it did so the way agencies handle things they would rather not explain: quietly, two days after walking his predecessor out the door. Kumar was a branch chief, a competent functionary several rungs below the job he was suddenly asked to do. This week, STAT reported, he is leaving too. That makes three. In roughly twelve months, the office that reviews the riskiest medicine the country makes has lost its director, her deputy, and the man brought in to replace them both.
The office is the Center for Biologics Evaluation and Research’s Office of Therapeutic Products, the one that reviews cell and gene therapies, among the most expensive treatments in medicine. Its director, Nicole Verdun, spent nearly two years building the modern framework for reviewing those products before she was placed on administrative leave in June 2025. Her deputy, Rachael Anatol, was walked out the same day. Kumar was named to replace them two days later, an emergency appointment dressed up as a succession. Now he is gone too.
The floor above has been just as porous, and the through-line is the same: the people who carry the institutional memory and the people who sign the accountable decisions are both vanishing. Peter Marks, who ran CBER for a decade and championed a fast, flexible approach to gene-therapy approvals, resigned under pressure in March 2025. His successor, Vinay Prasad, resigned in July 2025, came back roughly two weeks later at the agency’s request, and left again in the spring of 2026, his second exit described in an internal email as a “sabbatical.” Commissioner Marty Makary resigned in May 2026. By then both CDER and CBER, the two centers that decide what drugs and biologics reach Americans, were running on acting leadership at the same time.
You could call some of this housecleaning, and in part it is. The record on Verdun is more complicated than the press releases let on, because she was one of the reviewers Marks overruled in 2024 when he reached past his own staff to broadly approve Sarepta’s Elevidys, a gene therapy for Duchenne muscular dystrophy. That is worth sitting with. The scientists who urged caution were the ones later shown the door, and the man who waved the product through is gone as well, leaving behind a therapy whose safety record reads exactly like the thing the cautious reviewers were paid to worry about.
Here is what the flexibility bought. After Elevidys reached the market, the FDA suspended Sarepta’s distribution and placed several gene-therapy trials on hold after 3 deaths, all from acute liver failure, in non-ambulatory patients given Sarepta gene therapies. The agency then narrowed the label so the therapy could be used only in boys who can still walk. Marks had overruled the staff who told him to slow down. The patients who died of liver failure could not overrule anything.
The deaths are not the only reason the field has stopped congratulating itself. In May, Nature ran an investigation under a headline that would have been heresy during the boom years, asking whether a boy’s life-saving gene therapy had caused his brain tumour. The question is not hysteria. The oldest known hazard in this branch of medicine is insertional mutagenesis: a therapy that splices new genetic material into a patient’s chromosomes can, by the bad luck of where it lands, switch on a cancer gene. It is the risk that gave children leukemia in the earliest gene-therapy trials two decades ago, and it never went away. It was merely outshouted by the marketing.
So who, exactly, is supposed to be holding the line, and the honest answer right now is nobody in particular. The churn is not random. Fierce Pharma reported that Prasad departed amid conservative criticism and the controversy over his handling of Sarepta, after he moved to hold the company to account; read against the timeline, the reviewer who slowed a product down was removed faster than the product that preceded three deaths. The critics who wanted him gone were not exactly disinterested parties. When that is the sequence, it is not reform. It is capture wearing reform’s clothes.
None of this is an argument for the old regime. Marks’s version of “flexibility” is the failure this publication has catalogued for years: an FDA that treats the companies it regulates as its clients and patients as an acceptable margin of error. The tragedy of the moment is that the cure on offer is not competence. It is vacancy. The names on the door change, the lobbying does not, and the one constant is that the office meant to be the adult in the room on the most dangerous medicine in development keeps ending up empty.
Vijay Kumar never asked to be that adult. He was a branch chief handed an impossible job during a purge, and a year later he has done the rational thing and left. The office he is vacating will get another acting director, and probably another after that. The patients being injected with these therapies, meanwhile, do not get an acting body.
Sources
- STAT – FDA’s top gene therapy regulator is leaving his role (Vijay Kumar exits the Office of Therapeutic Products)
- BioPharma Dive – Gene therapy faces fresh uncertainty as Nicole Verdun and Rachael Anatol leave the FDA
- FDA – Requests Sarepta suspend distribution of Elevidys and places clinical trials on hold following 3 deaths
- FDA – New safety warning and revised indication limiting Elevidys to ambulatory patients after fatal liver injury
- Fierce Pharma – Vinay Prasad departs FDA amid conservative criticism and the Sarepta gene therapy controversy
- NPR – FDA Commissioner Marty Makary resigns after tumultuous tenure
- Pink Sheet / Citeline – Branch Chief Vijay Kumar Named Acting Head of US FDA’s Cell, Gene Therapy Office
- Clinical Trial Vanguard – FDA’s May 2026 leadership exodus: simultaneous CDER and CBER vacancies
- Nature – Did a boy’s life-saving gene therapy cause his brain tumour?