New York, March 9, 2026, 09:36 EDT
- uniQure shares surged in U.S. premarket action after news broke of FDA biologics chief Vinay Prasad’s planned departure.
- uniQure’s Huntington’s gene therapy ran into a wall with the FDA, which insisted on a fresh sham-surgery trial—sparking a tough battle before this latest move.
- Analysts noted the agency’s tone could be shifting, though the regulatory outlook remains up in the air.
uniQure shares surged roughly 34% to $14.27 in U.S. premarket action on Monday, following reports that the FDA’s vaccines and biologics chief plans to exit in April. The stock had already leapt 57% after hours on Friday after the initial report. Reuters
This goes beyond a staffing shuffle. uniQure is scrambling to keep its U.S. submission for AMT-130 alive. The FDA has told the company its Phase 1/2 results, stacked up against an external control arm, won’t cut it for a marketing application.
FDA Commissioner Marty Makary posted on X that the agency plans to announce Prasad’s replacement before his exit, saying Prasad was behind a number of policy shifts. During his time at the agency, Prasad faced criticism from both biotech leaders and patient advocates, with tensions surfacing over rare-disease drugs and gene therapy decisions involving companies like Sarepta Therapeutics and Moderna. Fierce Biotech
uniQure disclosed last week that the FDA has “strongly recommended” a prospective, randomized, double-blind study for AMT-130, this time using a sham surgery control. The gene therapy requires neurosurgical delivery. uniQure plans to request a follow-up meeting with regulators to talk through possible Phase 3 trial designs. Reuters
A few days on, strains intensified after a top FDA official—requesting anonymity—described AMT-130 as a “failed product” and took issue with uniQure’s approach to presenting its findings. uniQure, for its part, called those remarks “highly irregular” and insisted it still trusts its dataset. Reuters
uniQure wrapped up 2025 sitting on roughly $622.5 million in cash, cash equivalents, and current investment securities—enough, it estimates, to keep things running into the back half of 2029. CEO Matt Kapusta said the company still lacks “alignment” with the FDA on an approval pathway, though discussions with the agency will continue.
Stifel, in a note on Monday, called out uniQure as possibly the rare-disease drugmaker set to benefit most from Prasad’s exit. The firm also flagged several other gene therapy and rare disease names, noting the group has struggled under what it described as an “FDA risk off” environment. Investing.com
The leadership switch doesn’t solve the main issue: the FDA wants a fresh controlled trial, and uniQure’s therapy involves brain surgery—a controversy point over sham procedures in studies. The new chief might stick with this approach, or possibly take a tougher line.
Right now, investors are weighing the possibility of a reset at the FDA. The company faces the task of convincing regulators that its data is solid enough for a Biologics License Application—the standard route for U.S. approval of biologics like gene therapies.
