Key Takeaways
- Shares of uniQure (QURE) climbed 36% on Monday following the announcement that FDA’s Vinay Prasad will step down from the Center for Biologics Evaluation and Research
- Prasad had been responsible for overseeing uniQure’s Huntington’s disease treatment AMT-130 and faced allegations of overriding review teams
- RBC Capital moved QURE to Outperform from Sector Perform, increasing the price target to $35 from $11
- Other biotechs gained ground: REGENXBIO (RGNX) climbed 13% while Biohaven (BHVN) advanced 23%
- A Type B meeting with FDA is on uniQure’s calendar for Q2 2026
The past several days have delivered dramatic swings for uniQure investors. On March 5, an FDA representative publicly criticized the biotech firm for allegedly “performing a distorted or manipulated comparison” in its phase 1/2 study of AMT-130, a Huntington’s disease candidate. Surprisingly, shares still climbed 18% that session.
The following day, March 6, brought word that Vinay Prasad—who leads the FDA’s Center for Biologics Evaluation and Research (CBER)—plans to depart the agency in April for an academic position. QURE shares rocketed 34% higher that session, with Monday’s trading pushing gains to 36%.
Prasad had direct responsibility for regulatory decisions surrounding AMT-130. The agency informed uniQure that approval would not be granted based on comparisons to natural history data—a stance uniQure characterized as a “key shift” from prior agreements established before new FDA leadership took charge.
Additionally, the FDA demanded that the company conduct a comprehensive phase 3 trial, presenting significant financial and timeline obstacles for a rare disease therapy.
Investors and Wall Street analysts have recalibrated their outlook following Prasad’s resignation announcement. RBC Capital’s Luca Issi moved his rating on QURE to Outperform from Sector Perform while boosting his price objective from $11 to $35. His updated assessment places AMT-130’s approval probability at 50%.
“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
According to Stifel’s Paul Matteis, Prasad’s departure represents “a big win for biotech, especially for companies in the rare disease space.” He highlighted reports that Prasad had overturned internal FDA review teams to deliver unfavorable assessments on multiple therapies.
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 companies experienced unexpected changes to regulatory expectations that had been previously established during early-stage discussions with the agency, they noted.
The gene therapy sector broadly benefited from the announcement. REGENXBIO (RGNX) shares increased 13% while Biohaven (BHVN) jumped 23%.
Looking Ahead for uniQure
uniQure has scheduled a Type B meeting with FDA regulators for the second quarter of 2026. This discussion will prove critical as the company seeks clarity on AMT-130’s regulatory pathway forward.
Prasad’s official departure from the agency is scheduled for April.
Interestingly, on March 5—when the FDA official’s harsh criticism of uniQure’s study methodology became public knowledge—shares still managed an 18% gain. This suggested investors were already factoring in Prasad’s impact on the drug’s ultimate fate.
