Prasad to Step Down at FDA’s CBER

Rare Daily Staff

Vinay Prasad, the controversial head of the U.S. Food and Drug Administration’s Center for Biologics Evaluation and Research, which oversees vaccines and gene therapies, will leave the agency at the end of April.

The Wall Street Journal first reported Prasad’s planned departure. His tenure at the FDA was marked by clashes with industry and was a source of anxiety in the rare disease community. A number of rare disease therapies expected to reach the market failed to win approval on his watch, and the agency became less predictable in how it reviewed these drugs.

Prasad will return to his academic post at the University of California, San Francisco, where he has been a vocal critic of the FDA’s handling of COVID-19 vaccines and gene therapies.

The change at CBER comes as the agency rolls out its new framework for ultra-rare diseases.

FDA Commissioner Marty Makary, in a post on X, described Prasad’s government service as a “one-year sabbatical” and said the agency would name a successor before his departure. The stocks of several rare disease drug developers rose in after-hours trading Friday when the news became public.

Together with Makary, Prasad supported a new “plausible mechanism” framework that allows the FDA to rely more explicitly on mechanistic understanding—such as well-characterized genetic causality—when evaluating bespoke therapies for very small patient populations. Nevertheless, in case-by-case approval decisions, many rare disease drug developers and patient advocates felt he raised the bar for approval.

Photo: Vinay Prasad, head of FDA’s CBER