TLDR
- Shares of uniQure (QURE) climbed 36% on Monday following the announcement that FDA’s Vinay Prasad will depart from the Center for Biologics Evaluation and Research
- Prasad had been responsible for overseeing uniQure’s Huntington’s disease therapy AMT-130 and faced allegations of overruling internal review teams
- RBC Capital shifted QURE to Outperform from Sector Perform, boosting the price target from $11 to $35
- Related biotech stocks rallied: REGENXBIO (RGNX) climbed 13% and Biohaven (BHVN) jumped 23%
- The company is set for a Type B meeting with regulators in Q2 2026
The past week has been exceptionally volatile for uniQure shares. March 5 saw an FDA official publicly criticize the company for allegedly “performing a distorted or manipulated comparison” in its phase 1/2 study of AMT-130, an experimental Huntington’s disease therapy. Surprisingly, shares still climbed 18% by market close.
The following day, March 6, brought news that Vinay Prasad, who leads the FDA’s Center for Biologics Evaluation and Research (CBER), planned to depart the agency in April to pursue academic opportunities. QURE shares responded with a 34% gain that session. By Monday’s close, the stock had advanced 36%.
Prasad’s role included direct oversight of AMT-130’s regulatory review. The agency informed uniQure that approval wouldn’t be granted based solely on natural history comparisons — a stance the biotech firm described as a “key shift” from earlier agreements established before new FDA leadership took charge.
Regulators also demanded that uniQure conduct a comprehensive phase 3 trial, representing a significant financial and temporal obstacle for a rare disease candidate.
The leadership transition has clearly shifted investor and analyst sentiment. RBC Capital’s Luca Issi moved his rating on QURE to Outperform from Sector Perform, simultaneously raising his price objective from $11 to $35. His current approval probability estimate for AMT-130 stands at 50%.
“We believe that Prasad’s departure is likely to open up a more balanced discussion on risk/reward for HD,” Issi stated in his research note.
Analyst Reaction
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 suggesting Prasad had overridden internal FDA review teams to deliver unfavorable recommendations on multiple therapeutic candidates.
Truist’s research team observed that Prasad’s leadership “marked a sharp departure from the more flexible regulatory approach for rare and serious diseases” that characterized his predecessor Peter Marks’ tenure. Several biopharmaceutical companies, they emphasized, encountered evolving regulatory expectations despite earlier development discussions with the agency.
The gene therapy sector broadly benefited from the announcement. REGENXBIO (RGNX) advanced 13% while Biohaven (BHVN) surged 23% on the developments.
What’s Next for uniQure
The company has secured a Type B meeting with FDA regulators scheduled for the second quarter of 2026. This session will prove critical as uniQure works to establish a viable regulatory pathway for AMT-130.
Prasad’s official departure from the agency is slated for April.
Interestingly, on March 5 — the same day harsh criticism from an FDA official regarding uniQure’s trial methodology surfaced — shares still managed to close 18% higher, suggesting investors were already factoring in Prasad’s potential influence on the regulatory outcome.
Get 3 Free Stock Ebooks
Discover top-performing stocks in AI, Crypto, and Technology with expert analysis.
- Top 10 AI Stocks - Leading AI companies
- Top 10 Crypto Stocks - Blockchain leaders
- Top 10 Tech Stocks - Tech giants
