uniQure N.V. (NASDAQ:QURE) was halted pending news on Wednesday and appeared set for a strong move higher once trading resumed after announcing positive regulatory feedback from the U.S. Food and Drug Administration regarding its Huntington’s disease treatment candidate, AMT-130.
The company said the FDA has agreed that data from its ongoing Phase I/II study could form the primary foundation of a future Biologics License Application (BLA) seeking accelerated approval.
FDA Accepts Three-Year Study Data as Basis for Submission
According to uniQure, discussions with the regulator during a recent Type B meeting resulted in agreement that three-year follow-up data from the trial would be suitable as the principal dataset supporting a BLA filing.
The company plans to submit the application during the third quarter of 2026.
Management added that the FDA expressed a willingness to work closely and efficiently with uniQure to finalize details surrounding a post-approval confirmatory study before the BLA is filed.
Confirmatory Study Discussions Continue
The FDA is seeking further alignment on the design of the confirmatory trial that would support accelerated approval.
Among the topics under consideration is the potential use of a concurrent control group receiving standard-of-care treatment rather than employing a sham surgical procedure.
uniQure said it remains committed to launching the confirmatory study as quickly as possible and expects to continue discussions with the agency to finalize the protocol ahead of submission.
AMT-130 Holds Multiple FDA Designations
AMT-130 has already received several important regulatory designations from the FDA, including Regenerative Medicine Advanced Therapy (RMAT), Breakthrough Therapy and Fast Track status.
The RMAT designation was particularly significant as it represented the first time the FDA granted the designation to a treatment candidate targeting Huntington’s disease.
These designations are intended to facilitate the development and review of therapies addressing serious conditions with significant unmet medical needs.
Ongoing Studies Continue to Generate Data
uniQure is currently running two multicenter, dose-escalation Phase I/II studies evaluating the safety, tolerability and potential efficacy of AMT-130.
The U.S. trial enrolled 26 patients with early manifest Huntington’s disease, who were randomized to either treatment or a sham procedure.
Participants receiving therapy were administered a single dose of AMT-130 directly into the striatum using MRI-guided stereotactic neurosurgery.
Next Regulatory Milestone Approaches
The company expects to receive the official meeting minutes from the FDA within 30 days of the Type B meeting.
With a planned BLA submission targeted for the third quarter of 2026 and ongoing discussions around the confirmatory study design, investors are likely to focus on the next regulatory updates as AMT-130 advances toward a potential accelerated approval pathway.