uniQure plans Q3 2026 BLA filing for AMT-130 after FDA agrees 3-year data can support submission

The FDA has agreed that uniQure's existing 3-year Phase I/II clinical data for AMT-130, a gene therapy for Huntington's disease, can serve as the primary basis for a Biologics License Application seeking accelerated approval. This is a significant regulatory milestone, as it allows the company to proceed with a near-term BLA submission without requiring completion of a full pivotal trial first. The FDA also indicated it will work expeditiously with uniQure to align on the design of a required confirmatory study prior to BLA submission.

uniQure plans to file the Biologics License Application for AMT-130 in Q3 2026, which would be within the next few months. This timeline is contingent on aligning with the FDA on the confirmatory study design. A successful BLA submission and subsequent accelerated approval would allow the company to commercialize AMT-130 for Huntington's disease in the U.S. market ahead of completing the confirmatory study.

The FDA is requesting alignment on the design of a confirmatory study before the BLA is submitted. Notably, the FDA is considering allowing a concurrent control arm receiving standard-of-care therapy rather than a sham surgical procedure, which could make patient enrollment easier and address ethical concerns. uniQure has committed to conducting this confirmatory study without delay and expects to finalize the design with the FDA before filing the BLA.

AMT-130 holds three FDA designations intended to expedite development and review: Regenerative Medicine Advanced Therapy (RMAT), Breakthrough Therapy, and Fast Track. The RMAT designation is the first ever granted for Huntington's disease. These designations reflect the FDA's recognition of AMT-130's potential to address a serious unmet medical need and can facilitate more frequent interactions with the agency and potentially faster review timelines.

The Phase I/II program includes a U.S. study with 26 patients (16 treated, 10 sham control, plus 4 crossovers) and a European study with 13 patients. AMT-130 is administered as a single dose via stereotactic neurosurgery directly into the brain's striatum. The U.S. study had a 12-month blinded period followed by five-year follow-up. Additional cohorts explored combination with immunosuppression and treatment in patients with lower striatal volumes. These studies generated the 3-year data that the FDA has now agreed can support the BLA.

The company announced it will submit a Biologic License Application for AMT-130, its gene therapy candidate for Huntington's Disease, in Q3 2026. This represents a concrete regulatory milestone for the company's lead program.

The FDA indicated that 3-year data from the Phase I/II study would be sufficient to support a BLA for accelerated approval. This is a significant regulatory development that could expedite the path to market for AMT-130, though accelerated approval typically requires post-approval confirmatory studies.