FDA's Prasad Departs Amid Controversy Over Gene Therapy Regulation

In a sudden turn of events, Dr. Vinay Prasad has left his position as director of the FDA's Center for Biologics Evaluation and Research (CBER) after less than three months on the job. The departure comes amid a series of controversies surrounding gene therapy regulation and criticism from conservative figures.

Tumultuous Tenure at CBER

Prasad's brief stint at the FDA was marked by significant policy shifts and public disputes. As head of CBER, he oversaw the regulation of vaccines, cell therapies, and gene therapies. His appointment in May 2025 was initially hailed by FDA Commissioner Martin Makary as a "significant step forward" for the agency, citing Prasad's reputation for scientific rigor and independence.

During his tenure, Prasad quickly implemented stricter approval guidelines for COVID-19 vaccines and overruled agency reviewers multiple times to issue narrower-than-requested clearances for shots developed by Moderna and Novavax. These actions raised concerns among biotech companies and investors about potential shifts in FDA standards.

Sarepta Controversy and Conservative Backlash

The most high-profile incident of Prasad's tenure involved a regulatory tug-of-war with Sarepta Therapeutics over its Duchenne muscular dystrophy (DMD) gene therapy, Elevidys. Following patient deaths, the FDA demanded Sarepta halt all shipments of the therapy. However, in a surprising reversal, the agency allowed Sarepta to resume giving Elevidys to ambulatory DMD patients just days later.

This flip-flop coincided with mounting criticism of Prasad from conservative commentators. Right-wing activist Laura Loomer labeled Prasad a "leftist saboteur" undermining President Donald Trump's FDA agenda. The Wall Street Journal published op-eds characterizing him as a "Bernie Sanders acolyte" and a "one-man death panel."

Implications for Gene Therapy Regulation

Prasad's departure has sparked speculation about potential shifts in the FDA's approach to gene therapy regulation. Some analysts suggest it could signal a move towards a more permissive, "right-to-try" oriented stance on rare disease treatments.

William Blair analysts argued that Prasad's exit "may alleviate some of the overhang on the cell and gene therapy spaces" due to his previous criticism of the accelerated approval pathway. However, the impact on vaccine regulation remains uncertain, as FDA Commissioner Makary has expressed similar views on vaccine oversight.

As the pharmaceutical industry digests this latest development, questions remain about the future direction of CBER and its approach to innovative therapies. The abrupt nature of Prasad's departure leaves many wondering about the potential influence of political pressure on FDA decision-making and the delicate balance between regulatory caution and facilitating access to cutting-edge treatments.