uniQure has submitted a biologics license application (BLA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval of its Huntington’s disease gene therapy, AMT-130, also known as ifezuntirgene inilparvovec. The company has also filed an application for the therapy with the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA).
The submission follows months of regulatory disagreements surrounding the treatment and comes after the FDA reversed an earlier position that existing data were insufficient to support a marketing application.
Priority Review Request Could Lead to 2027 FDA Decision
Alongside the BLA, uniQure has requested priority review from the FDA. If granted, the designation would reduce the agency’s review period to six months rather than the standard 10 months. The company said that, if the application is accepted for priority review, the FDA could set an action date for potential accelerated approval in late April or early May 2027.
If approved, AMT-130 would be the first genetic treatment for Huntington’s disease.
In a statement announcing the filings, uniQure CEO Matt Kapusta said, “We are grateful to the FDA for its leadership in advancing regulatory science to meet the urgency of this disease, and to the MHRA for its commitment to advancing rare disease treatments in the U.K.”
Three-Year Trial Results Form Basis of Application
The applications are supported by three-year data from a Phase 1/2 trial that compared patients treated with AMT-130 with a propensity score-matched external control derived from the Enroll-HD natural history database.
Data announced in September 2025 showed a 75% slowing of disease progression compared with the matched external controls. The company has also said it expects to present four-year data later this quarter.
Huntington’s disease is a rare inherited brain disorder that causes movement difficulties, behavioral changes, and cognitive decline. According to the information provided, no approved drugs currently exist that help slow disease progression.
Regulatory Dispute Preceded Submission
The filing comes after a period of disagreement between uniQure and the FDA.
In November 2025, the FDA informed the company that it no longer agreed that data from the Phase 1/2 study were adequate to provide the primary evidence supporting a BLA submission. uniQure said the agency had previously agreed to the trial protocols and statistical analyses.
In February, then-FDA Commissioner Marty Makary appeared to criticize an unnamed therapy involving direct administration into the brain through a surgically created opening in the skull. Although he did not identify the product, the comments were widely believed to refer to AMT-130.
The FDA also faced criticism for seeking a new study that would have included a placebo-surgery control group, requiring some participants to undergo invasive brain procedures without receiving the gene therapy.
In June, the agency reversed its earlier position and agreed that the existing three-year data could support an application for accelerated approval.
Confirmatory Trial Discussions Continue
Analysts at William Blair said the Phase 1/2 trial design aligns with FDA publications and draft guidance indicating that external controls may be appropriate in certain gene therapy studies involving small patient populations.
The firm also stated that uniQure and the FDA have reached alignment on key elements of a confirmatory trial. According to analysts, the planned study would enroll around 200 patients and use 2:1 randomization between AMT-130 and standard of care.
Stifel similarly stated that the company expects the confirmatory study to be well underway before the FDA reaches a decision on accelerated approval.
uniQure has submitted regulatory applications in both the United States and United Kingdom for AMT-130, an investigational gene therapy designed to treat Huntington’s disease. The submissions represent a major regulatory milestone for uniQure and could potentially make AMT-130 the first genetic medicine approved for Huntington’s disease.
uniQure Submits U.S. BLA for AMT-130
uniQure submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for AMT-130. The company has also requested priority review, which could shorten the FDA review timeline if granted.
The submission follows a June 2026 Type B meeting in which the FDA communicated that three-year Phase I/II data could serve as the primary basis for an accelerated-approval application.
