FDA Center for Biologics Evaluation and Research director Vinay Prasad will leave the agency at the end of April, FDA Commissioner Marty Makary told The Wall Street Journal on Mar. 9. Analysts say his departure could have significant effects on biotech companies, especially those focused on rare diseases.
Prasad's exit is seen as a potential turning point for drugmakers that have faced regulatory hurdles under his leadership. Stifel analysts wrote in a note to investors that the move "is a big win for biotech, especially for companies in the rare disease space." They highlighted uniQure as a top beneficiary, noting the company's ongoing efforts to advance its gene therapy AMT-130 for Huntington’s disease.
In November 2025, the FDA informed uniQure it no longer agreed that its Phase 1/2 study was sufficient for a drug application, despite earlier indications of support. This led to public disputes between uniQure and the regulator. Last week, an unnamed senior FDA official stated during a press call that while patients would not need placebo burr holes drilled into their skulls during trials, they would undergo anesthesia and receive minor scalp incisions instead. Stifel analysts commented that "AMT-130 has evolved into a symbol of FDA inflexibility, or at the very least FDA inconsistency," adding that Prasad’s departure increases the likelihood of uniQure receiving a product review as previously expected.
The news of Prasad's planned departure coincided with notable stock gains for several biotech firms. UniQure's shares rose by 51% in premarket trading Monday after closing at $14.27 Friday; Replimune also saw its stock rise nearly 16%. Replimune had received an unexpected rejection from the FDA in July 2025 for its RP1 treatment for advanced melanoma—a decision BMO Capital Markets said was made without prior concerns raised during mid-cycle reviews. Disc Medicine experienced a smaller increase after failing to secure approval last month for bitopertin to treat erythropoietic protoporphyria; reports indicated Prasad had been skeptical about this therapy internally.
Other companies potentially benefiting from this leadership change include Capricor Therapeutics, REGENXBIO, and Lexeo Therapeutics according to Stifel analysts. However, they cautioned that uncertainty remains regarding who will replace Prasad: “Will it be someone with actual regulatory experience or a regulatory track record?”
RBC Capital Markets suggested several possible candidates based on previous analyses and connections with Commissioner Makary. These include infectious disease expert Stefan Baral—who shares Makary’s views on COVID-19 vaccine mandates—and others such as cancer specialist Jin He, vascular surgeon Caitlin Hicks, and psychiatric expert Chris Palmer.
