FDA Rejects uniQure’s AMT-130 Data, Demands Sham-Controlled Phase III Study
FDA rejected uniQure’s Phase I/II and external-control data for AMT-130, demanding a prospective, randomized, sham-controlled Phase III trial, prompting a 41% share plunge to $9.19 and cutting market cap to $613.6 million. uniQure will request a Type B meeting in Q2 2026 to discuss prospective Phase III study design.
1. FDA Demands Additional Study
The FDA held a Type A meeting with uniQure and determined that Phase I/II trial results for AMT-130, relying on external control comparison, do not meet primary effectiveness evidence standards. The agency strongly recommended a prospective, randomized, double-blind, sham surgery-controlled Phase III trial before marketing approval can proceed.
2. Share Price Impact and Market Cap
Following the FDA announcement, uniQure’s Nasdaq-listed shares tumbled 41% from $15.63 at the close on February 27 to $9.19 at the open on March 2. The market capitalization decreased to approximately $613.6 million, reflecting investor concern over the extended development timeline and potential costs.
3. Next Steps and Regulatory Timeline
uniQure plans to request a Type B meeting in Q2 2026 to discuss the prospective trial design with the FDA. Meanwhile, the company continues dialogue on Phase III development and must also address a separate FDA-mandated pause on its Fabry disease program initiated in February 2026.