Key Highlights
- uniQure (QURE) shares climbed 36% Monday following Vinay Prasad’s announcement that he will leave the FDA’s Center for Biologics Evaluation and Research
- Prasad directed the regulatory review of uniQure’s Huntington’s disease therapy AMT-130 and faced allegations of overturning review team recommendations
- RBC Capital moved QURE to Outperform from Sector Perform, increasing the price target from $11 to $35
- Other biotech stocks rallied as well: REGENXBIO (RGNX) climbed 13% while Biohaven (BHVN) added 23%
- A Type B meeting between uniQure and the FDA is planned for Q2 2026
uniQure has experienced remarkable volatility in recent trading sessions. On March 5, an FDA official publicly stated the company had performed a “distorted or manipulated comparison” in its phase 1/2 clinical trial for AMT-130, a Huntington’s disease treatment. Despite this criticism, shares rose 18% by market close.
The following day, March 6, investors learned that Vinay Prasad, who leads the FDA’s Center for Biologics Evaluation and Research (CBER), would depart the agency in April to rejoin academic medicine. QURE surged an additional 34% that session. By Monday’s close, the total gain reached 36%.
Prasad held direct oversight of AMT-130’s regulatory review process. The FDA informed uniQure that approval would be denied based on the company’s comparison to natural history data — a stance uniQure described as a “key shift” from prior agreements made before new leadership took control at the agency.
The regulatory body also demanded that uniQure conduct a complete phase 3 clinical trial, representing a significant financial and timeline burden for a therapy targeting a rare condition.
The announcement of Prasad’s exit has reshaped the outlook among investors and Wall Street analysts. RBC Capital’s Luca Issi moved his rating on QURE to Outperform from Sector Perform while raising his price target from $11 to $35. His updated model assigns a 50% probability to eventual AMT-130 approval.
“We believe that Prasad’s departure is likely to open up a more balanced discussion on risk/reward for HD,” Issi wrote.
Wall Street Weighs In
Stifel’s Paul Matteis characterized Prasad’s departure as “a big win for biotech, especially for companies in the rare disease space.” He highlighted reports that Prasad had overturned internal FDA review team decisions to issue unfavorable recommendations on multiple therapies.
Analysts at Truist observed that Prasad’s leadership “marked a sharp departure from the more flexible regulatory approach for rare and serious diseases” that characterized the tenure of his predecessor, Peter Marks. Several companies encountered changing regulatory expectations that contradicted earlier agreements established during development discussions with the agency.
The gene therapy sector broadly benefited from the announcement. REGENXBIO (RGNX) advanced 13% while Biohaven (BHVN) posted a 23% gain.
The Road Ahead for uniQure
uniQure has scheduled a Type B meeting with the FDA during the second quarter of 2026. This session will serve as a critical opportunity for the company to determine the regulatory pathway for AMT-130.
Prasad will formally depart the agency in April.
On March 5, when the FDA official’s harsh assessment of uniQure’s trial methodology became public knowledge, shares still finished the day 18% higher — indicating that markets were already factoring in Prasad’s role in the drug’s regulatory fate.
