Shares of uniQure tumbled after the FDA signaled it would require a sham-controlled Phase 3 trial for the company's Huntington's disease therapy, AMT-130, significantly delaying its potential approval. The regulatory decision came after a Type A meeting where FDA officials "strongly" recommended a prospective sham-controlled study rather than the more flexible approach uniQure had hoped for.

Mizuho analysts slashed their price target to $12 from $33 and downgraded the stock to Neutral, citing diminished confidence in regulatory flexibility. They pushed the expected launch to 2030 from 2028 and reduced the probability of success to 15% from 65%. BofA Securities also downgraded shares to Sector Perform with a $11 price target, noting the FDA's unwillingness to approve AMT-130 based on natural history data comparisons.

Both firms emphasized that ongoing Phase 1/2 data, including a four-year readout expected in Q3 2026, are unlikely to support approval under the current regulatory stance. The broader pipeline faces challenges, with the SOD1-ALS study on hold after a serious adverse event and mid- to high-dose Fabry disease cohorts paused. The sham-controlled requirement raises ethical concerns and execution difficulties that could fundamentally alter uniQure's development timeline.