uniQure N.V.’s (NASDAQ:QURE) shares surged after news that Dr. Vinay Prasad will step down from leading the U.S. Food and Drug Administration’s biologics division.
On Friday, FDA Commissioner Marty Makary confirmed that Dr. Vinay Prasad, head of the Food and Drug Administration’s (FDA) Center for Biologics Evaluation and Research (CBER), is set to leave his position at the end of April.
FDA’s CBER is responsible for evaluating a wide spectrum of treatments, including cancer drugs, gene therapies, and vaccines.
Controversial Regulatory Decisions Mark Prasad’s Tenure
His tenure was marked by several controversial decisions, including regulatory reversals for rare-disease drugs, including uniQure’s gene therapy, and a refusal to consider a flu shot application from Moderna Inc. (NASDAQ:MRNA)
In May 2025, the FDA Commissioner named Dr. Vinay Prasad the next director of CBER. Dr. Prasad had stepped down on July 30.
The FDA rehired Prasad in August 2025.
Dr. Prasad had railed against a decision to approve Sarepta Therapeutics Inc.’s (NASDAQ:SRPT) drug for Duchenne muscular dystrophy known as Elevidys, arguing that there was little evidence favoring the gene therapy.
Criticism Over uniQure’s Huntington’s Disease Drug Rejection
More recently, Prasad has come under fire for the FDA’s rejection of uniQure’s Huntington’s disease gene therapy.
The FDA advised against using Phase 1/2 study data as the primary evidence for the marketing application of AMT-130, raising uncertainty around the therapy’s regulatory path.
The agency recommended that uniQure conduct a prospective, randomized, double-blind, sham surgery-controlled study to proceed with its application.
Last week, an FDA official defended the request for an additional study.
Ongoing Debate Over Placebo-Controlled Trials in Rare Diseases
The Wall Street Journal, first to report Prasad’s departure, highlighted that UniQure’s trial did not include a placebo control arm.
In 2024, the FDA said the company could still pursue accelerated approval without one, citing the challenges of enrolling patients with a rare, debilitating disease into placebo-controlled studies.
The agency reiterated this view in guidance issued last week. uniQure’s therapy also requires brain surgery, making it unlikely that patients would enroll in a trial where they might receive a placebo.
Makary told CNBC that “we [the FDA] do not believe that you need to use a randomized controlled trial [RCT] model for all rare diseases. We believe in regulatory flexibility.”
uniQure Shares Surge on Departure News
uniQure shares surged 31.39% to $18.75 in premarket trading Monday, according to Benzinga Pro data, extending momentum after the stock jumped 33.99% on Friday to close at $14.27.
A Bloomberg report said uniQure shares jumped as much as 70% in post-market trading following news of Prasad’s departure. The company has been in a public dispute with his division over regulatory requirements for its Huntington’s disease gene therapy.
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