Key Takeaways
- FDA leadership shift underscores regulatory volatility: Vinay Prasad’s resignation follows high-profile drug application rejections and safety-related restrictions, signaling increased scrutiny regarding gene therapies and accelerated approvals.
- Sarepta’s Elevidys under intensified safety review: FDA's initial pause of the shipments for the drug and subsequent clearance for ambulatory patients highlight evolving regulatory expectations for gene therapies after multiple patient deaths.
- Complete Response Letters (CRLs) raise concerns over FDA communication practices: Recent CRLs issued to Replimune and Capricor suggest possible gaps in agency-sponsor dialogue during mid- and late-cycle reviews.
Vinay Prasad, head of the FDA’s Center for Biologics Evaluation and Research (CBER), has resigned after a series of contentious decisions, including rejections of Biologics License Applications (BLAs) for rare disease therapies and enforcement of product restrictions. Prasad leaves at a time in which criticism of his short tenure grew across the political landscape, including from conservative media figures such as former US Senator Rick Santorum and influencer Laura Loomer.1
Why Did the FDA’s Top Biologics Official Step Down?
"Dr. Prasad did not want to be a distraction to the great work of the FDA in the Trump administration and has decided to return to California and spend more time with his family," a Department of Health and Human Services spokesperson said in an emailed statement to Reuters. “We thank him for his service and the many important reforms he was able to achieve in his time at FDA.”1,2
Leadership and Background
First announced as the head of CBER in May, Prasad was tasked with overseeing vaccines and biologic therapies and later assumed the position of the FDA’s chief medical and scientific officer. He replaced Peter Marks, who was forced out in March amid disagreements with Health Secretary Robert F. Kennedy Jr. Similar to other health officials appointed under the new administration, Prasad had been openly critical of the federal response and vaccination strategies during the COVID-19 pandemic.3
Sarepta and the Elevidys Controversy
Notably during Prasad’s tenure, the FDA placed restrictions on Sarepta Therapeutics’ gene therapy, Elevidys, due to multiple deaths in patients taking the drug. On July 18, 2025, the FDA requested that Sarepta suspend all Elevidys shipments and placed all limb-girdle muscular dystrophy (LGMD) gene therapy trials on clinical hold, citing safety concerns. While initially declining to stop shipments—stating that no new safety signals had emerged during trials—Sarepta’s stock fell by 36% in one day in the face of FDA pressure and the announcement of a third reported death. A few days later, the company reversed course and halted shipments.4