Some biotechs that had seen regulatory setbacks under Center for Biologics Evaluation and Research director Vinay Prasad experienced stock bumps Monday morning. Under Prasad’s leadership, the rare disease space has suffered a series of controversial rejections.
Depending on who replaces him, rare disease drugmakers stand to win big from the upcoming departure of FDA Center for Biologics Evaluation and Research director Vinay Prasad, Stifel analysts said Friday. The oncologist has been a nexus of controversy for the agency.
Prasad will leave the FDA at the end of April, FDA Commissioner Marty Makary told The Wall Street Journal on Friday. His departure, analysts at Stifel wrote in a Sunday note to investors, “is a big win for biotech, especially for companies in the rare disease space.”
At the top of Sifel’s list of winners is uniQure, which recently has been “getting so much attention in the public press and political realm.” The biotech is advancing a gene therapy called AMT-130 for the treatment of Huntington’s disease. In November 2025, the FDA told uniQure it “no longer agrees” that the company’s Phase 1/2 study is sufficient to warrant a drug application—despite allegedly signing off on the program months earlier.
The episode kicked off a public back-and-forth between uniQure and the regulator, culminating in an FDA press call last week where an unnamed senior official at the agency blasted the biotech and insisted that the FDA’s request for a sham-controlled trial is both feasible and ethical.
“AMT-130 has evolved into a symbol of FDA inflexibility, or at the very least FDA inconsistency,” Stifel wrote on Sunday. And while Prasad’s exit doesn’t guarantee a favorable verdict for the gene therapy, it does make it more likely that uniQure “will get the product review that they were seemingly promised,” the analysts continued.
At $21.55, uniQure is up 51% in premarket trading on Monday. Its closing price on Friday, shortly before the news of Prasad’s departure broke, was $14.27. BioSpace has reached out to both uniQure and the FDA for comment.
Also enjoying a healthy stock bump is Replimune, which hit $9.35 per share before Monday’s opening bell, up nearly 16% from its closing price of $8.07 on Friday. The biotech in July 2025 received a surprise rejection for its viral immunotherapeutic asset RP1 for advanced melanoma, a decision that prompted more than 20 experts in the field to urge the FDA to re-review its verdict.
“When Replimune initially filed for approval of RP1, no concerns were raised with their application during mid-cycle reviews, but the company was subsequently handed a CRL,” analysts at BMO Capital Markets wrote on Friday, although it added that the level of involvement from Prasad in this decision was “unclear.”
BMO also highlighted Disc Medicine, which last month failed to secure regulatory approval for bitopertin for the rare disease erythropoietic protoporphyria. Last year, news circulated that Prasad had internally been skeptical of bitopertin. Disc enjoyed a small bump of 1.34% to $62 apiece in premarket trading on Monday.
Other potential gainers from Prasad’s departure include Capricor Therapeutics, REGENXBIO and Lexeo Therapeutics, according to Stifel. In its Sunday note, however, the firm conceded that the question of Prasad’s replacement remains a key overhang for the rare disease space.
“Will it be someone with actual regulatory experience or a regulatory track record?” the analysts asked.
RBC Capital Markets offered a few potential candidates. In a Friday note, the firm pointed to its own May 2025 analysis, suggesting prominent people that Makary could tap as CBER director. The analysts in particular looked at Makary’s “most frequent co-authors” and “those with ties to him on social media.”
One potential candidate could be Stefan Baral, who is an infectious disease expert from the Johns Hopkins School of Public Health and shares Makary’s skepticism of COVID-19 vaccine mandates and the “financial conflicts of interest” in biopharma, according to RBC. The group offered many other potential candidates, including cancer specialist Jin He, vascular surgeon Caitlin Hicks and psychiatric expert Chris Palmer.
