BLA filed for Huntington's gene therapy on an externally controlled 36-month efficacy analysis; FDA's willingness to approve that evidence package is the only question that matters

uniQure submitted BLA and MAA for AMT-130 (ifezuntirgene inilparvovec) in Huntington's disease with priority review request, planning four-year Phase I/II data presentation and Type B FDA meeting on confirmatory trial design.

The BLA and MHRA MAA are filed on schedule, and FDA confirmed in writing in July that pursuing the accelerated-approval pathway on externally controlled data is a reasonable approach -- that keeps the submission pathway viable, not settled: FDA acceptance of the BLA for filing, MHRA validation of the MAA, and ultimately whether the evidence package itself is deemed sufficient for approval are all still open questions the company's own disclosures continue to flag as at-risk.

FDA Type B meeting officially confirmed accelerated approval pathway and BLA submission strategy for AMT-130, clearing regulatory pathway uncertainty for Q3 2026 submission.

FDA's official written meeting minutes, disclosed in the Q2 filing, confirm the three-year single-arm data can support an accelerated-approval BLA submission, resolving the uncertainty that had kept the June reversal unconfirmed -- though discussions to align on the confirmatory study design remain ongoing.

FDA verbally indicated 3-year AMT-130 data could support an accelerated approval BLA, but confirmatory trial design remains unaligned and written meeting minutes are still pending; uniQure plans Q3 2026 submission.

FDA verbally indicated at a Type B meeting that the three-year single-arm data could support an accelerated-approval BLA, reversing January's rejection -- but confirmatory study design remains unaligned and the company has not yet received the meeting's written minutes, so the reversal is not yet a confirmed regulatory position.