uniQure's Huntington's Disease Gene Therapy AMT-130 Remains on Track for Historic Q3 2026 BLA Submission Under Accelerated Path
Following a productive Type B meeting with the U.S. Food and Drug Administration (FDA) and receipt of official meeting minutes in July 2026, uniQure has confirmed that it remains on track to submit its Biologics License Application (BLA) for AMT-130 in the third quarter of 2026. The BLA will seek accelerated approval for the investigational gene therapy as a potential first-in-class, disease-modifying treatment for Huntington's disease (HD).
Accelerated Approval Pathway and Propensity-Matched Controls
The FDA has agreed that uniQure's existing 3-year Phase I/II clinical data can support a BLA submission under the accelerated approval pathway.1 The primary efficacy analysis will compare changes in the composite Unified Huntington’s Disease Rating Scale (cUHDRS) in patients treated with high-dose AMT-130 against a propensity score-matched external control derived from the ENROLL-HD natural history dataset. At 3 years, high-dose AMT-130 demonstrated a 75% slowing of disease progression on the cUHDRS compared to these matched controls.
Confirmatory Study Design and Regulatory Alignment
A critical component of uniQure's ongoing discussions with the FDA is the design of the post-approval confirmatory study. The FDA has indicated that the confirmatory study must be well underway, and potentially fully enrolled, at the time of accelerated approval.
To ensure feasibility and patient enrollment, the FDA and uniQure are aligning on a randomized, concurrent standard-of-care control design instead of a sham surgical procedure. This is a major structural shift that is expected to significantly accelerate the confirmatory trial's timeline and improve patient recruitment. uniQure plans to initiate the confirmatory study as expeditiously as possible after aligning on the final trial design.
Near-Term Milestones and Financial Strength
uniQure's regulatory momentum is backed by a robust cash runway. Following a $259 million public offering in July 2026, the company ended Q2 2026 with $810.3 million in cash and cash equivalents, which is projected to fund operations into 2030 despite a Q2 net loss of $81.1 million.
In addition to the Q3 2026 BLA submission in the United States, uniQure is progressing with a parallel regulatory submission to the UK's Medicines and Healthcare products Regulatory Agency (MHRA) in Q3 2026. The company also plans to release highly anticipated topline four-year clinical data from the AMT-130 Phase I/II program in September 2026.
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An instance of Accelerated pathways shift the proof of clinical benefit from pre-market trials to post-marketing requirements. — The regulatory framework allows a gene therapy developer to file for market access using early phase data, shifting the definitive proof of efficacy to a post-approval confirmatory study. ↩︎