uniQure to file AMT-130 BLA for Huntington's disease in Q3 2026
uniQure has announced that the FDA, during a recent Type B meeting, confirmed that three-year data from the company's Phase I/II study of AMT-130 can serve as the primary basis for a Biologics License Application under the accelerated approval pathway. The NASDAQ-listed gene therapy company intends to file the BLA in the third quarter of 2026, a milestone that would represent one of the most significant regulatory advances in Huntington's disease research to date.
AMT-130 is delivered as a single administration via MRI-guided, convection-enhanced stereotactic neurosurgery directly into the striatum. The US Phase I/II study enrolled 26 patients with early manifest Huntington's disease, randomised to low dose, high dose, or a sham procedure. A parallel European open-label study enrolled 13 patients. The FDA has agreed that data from treated cohorts can be compared against a propensity score-matched external control drawn from the Enroll-HD natural history dataset, under a prespecified statistical analysis plan.
Regulatory path
A condition of accelerated approval is the conduct of a confirmatory study. The FDA has indicated it wishes to align on that study's design before the BLA is submitted, and has signalled openness to using a concurrent standard-of-care control arm rather than the sham procedure employed in the Phase I/II programme. uniQure said it expects to receive formal meeting minutes within 30 days and has committed to initiating the confirmatory study without delay.
AMT-130 holds three FDA expedited designations: Regenerative Medicine Advanced Therapy (RMAT), the first RMAT granted for Huntington's disease; Breakthrough Therapy; and Fast Track. These designations provide rolling review rights and intensive FDA engagement, which will be material as uniQure works to finalise the confirmatory study protocol ahead of submission.
Chief executive Matt Kapusta said the consistency and strength of clinical data generated to date gave the company "great confidence in the product's potential to make a meaningful difference for patients," adding that AMT-130 would be brought to US and global patients "as quickly and responsibly as possible."
Market context and competitive landscape
Huntington's disease affects approximately 75,000 people across the US, EU and UK, with no currently approved therapy capable of slowing disease progression. The condition's clear genetic aetiology, a CAG repeat expansion in the huntingtin gene, has long made it an attractive target for gene-silencing and gene-replacement strategies, but clinical translation has proved challenging. Wave Life Sciences and Roche have both advanced antisense oligonucleotide programmes in the space, with mixed Phase 3 outcomes, underlining the difficulty of the indication and the significance of uniQure's regulatory progress.
If the BLA is accepted and accelerated approval is granted, AMT-130 would enter a market with substantial unmet need and limited competition, though the surgical delivery route and single-treatment model will require significant infrastructure development at treatment centres. Pricing and reimbursement for one-time gene therapies remain a broader sector challenge; recent precedents in haemophilia and spinal muscular atrophy suggest list prices at or above seven figures, with payers increasingly demanding outcomes-based contracts. Investors will watch closely for the confirmatory study design, which will shape both the post-approval commercial timeline and the long-term label.