This biotech's stock soared after a 'polarizing' FDA official is stepping down

By Jaimy Lee

Shares of UniQure, which is developing a gene therapy for Huntington's disease, are rallying

Vinay Prasad, an official for the Food and Drug Administration, is stepping down.

A controversial political appointee at the Food and Drug Administration plans to step down for a second time, and the biotech company at the center of a recent debate that involved him saw its stock soar.

UniQure's stock (QURE) closed 26% higher on Monday. Late Friday, the FDA told the Wall Street Journal that Vinay Prasad plans to leave the agency next month. He is the director of the FDA's Center for Biologics Evaluation and Research, which oversees vaccines and rare-disease drugs. A FDA spokesperson confirmed his departure.

Prasad had reportedly been involved in the decision to request a new trial for uniQure's experimental gene therapy for Huntington's disease. UniQure's stock plunged on that request, falling 33% in a single day, and the action led to a broader discussion about the expertise of current FDA leadership and how rare-disease-focused biotechs have struggled to advance therapies under those leaders.

Wall Street welcomed the news of Prasad's departure, though some analysts cautioned against too much excitement without knowing who his replacement will be.

"Constructive engagement with sponsors while maintaining scientific rigor would be a welcome respite," Leerink Partners analyst Joseph Schwartz told investors on Sunday. "Net-net, we expect sentiment to improve materially."

UniQure said earlier this month that the FDA had recommended the company run a new Phase 3 trial for its gene therapy that includes a controlled sham surgery. It was surprised by the request for a new trial and the inclusion of sham surgery for the placebo group. The experimental therapy is administered through a catheter inserted into tiny holes in the skull. However, the idea of drilling holes in patients for a placebo is widely considered unethical, including by regulators in Europe, according to Schwartz.

Regenxbio (RGNX), another rare-disease biotech that recently sparred with the FDA, also saw its shares close higher on Monday, about 20% over Friday's closing price. The regulator had declined to approve its experimental treatment for Hunter syndrome based on concerns with the external control group. Moderna (MRNA) has had its own challenges getting the FDA to review its mRNA-based flu shot. The agency refused to review the vaccine candidate and then backtracked to allow the submission.

"Prasad was a polarizing figure as exemplified by his involvement in rare-disease drug regulatory reversals, an initial refusal (and rapid reversal) to review Moderna's flu-shot application after the company agreed to additional confirmatory data, and internal personnel complaints," Raymond James analyst Chris Meekins wrote in a note on Monday. "His actions created significant political and operational friction for the administration."

UniQure's stock is down 39% so far this year through Friday, while the biotech index XBI has gained 2%.

-Jaimy Lee

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03-09-26 1638ET