Overview
- Final FDA minutes from an October pre-BLA meeting confirm the agency views AMT-130’s current Phase I/II data as insufficient as primary evidence for a filing.
- uniQure said it will urgently request a follow-up meeting with the FDA in the first quarter of 2026 to discuss a viable regulatory path.
- Shares fell again on the update, with the stock down roughly 11% to 16% during Thursday trading following steep losses reported in early November.
- AMT-130 is designed to curb production of mutant huntingtin protein, and a September readout reported about 75% slowing of progression in 12 high-dose patients versus an external control over three years.
- Analysts outline potential paths that range from additional patients or longer follow-up to a new confirmatory trial, with some still seeing a chance for a negotiated route.