uniQure FDA Advises Sham-Controlled AMT-130 Study, Analysts Cut Targets to $9 and $15
uniQure shares rose after an FDA recommendation against using Phase 1/2 data for its AMT-130 Huntington’s gene therapy application, advising a prospective, randomized, double-blind, sham-controlled study instead. Analysts slashed price targets—Goldman Sachs to $9, Wells Fargo to $15—while shares trade near $9.16, close to 52-week lows.
1. FDA Recommends New Controlled Study
The U.S. regulatory authority advised uniQure against relying on Phase 1/2 data as the primary evidence for its AMT-130 marketing application and strongly recommended a prospective, randomized, double-blind, sham surgery-controlled trial to support any future Biologics License Application.
2. Regulatory Impact and Next Steps
In November 2025, uniQure believed its Phase 1/2 data might suffice for a BLA submission, but the recent guidance raises uncertainty around AMT-130’s approval timeline; the company plans to request a Type B meeting in Q2 2026 to discuss study design and data requirements.
3. Stock Reaction and Analyst Outlook
Shares rebounded modestly to $9.16 after a sharp prior-session drop, trading near 52-week lows as technicals show a bearish trend below key moving averages; analysts responded by cutting price targets—Goldman Sachs to $9, Wells Fargo to $15, and Chardan Capital to $16—reflecting heightened regulatory risk.