July 29, 2026 —
uniQure said the regulatory path for AMT-130, its investigational gene therapy for Huntington’s disease, has become clearer following recent alignment with the U.S. Food and Drug Administration, though the company expects it may need to participate in an FDA advisory committee meeting.
AMT-130 is designed as a potential first disease-modifying therapy for Huntington’s disease, a fatal inherited neurodegenerative disorder. If approved, it would be the first therapy indicated for Huntington’s disease.
uniQure Chief Medical Officer Walid Abi-Saab said on the company’s quarterly earnings call that an advisory committee meeting is likely, and that the company is preparing for the possibility. FDA advisory committees are often used when the agency is reviewing first-in-class therapies, products for diseases with no approved treatments, or programs supported by novel datasets.
AMT-130 has had a complex regulatory path. In November 2024, the FDA declined to accept uniQure’s filing, citing insufficient data from the company’s Phase 1/2 study. After a January meeting, uniQure said the agency had requested another study.
Following a subsequent meeting in June, the FDA shifted its position and indicated that three-year data from the Phase 1/2 study could support a Biologics License Application under the accelerated approval pathway. The company now plans to move forward with license application submissions in the United States and the United Kingdom.
Before the BLA submission, uniQure and the FDA still need to align on the design of a confirmatory study. Under current accelerated approval expectations, confirmatory studies should generally be underway, and ideally fully enrolled, by the time accelerated approval is granted.
uniQure said it plans to begin the confirmatory study as quickly as possible once the design is agreed with the FDA. The study will be important for verifying clinical benefit after any potential accelerated approval.
The company also plans to present updated data from its ongoing Phase 1/2 AMT-130 studies in September 2026. The update is expected to include four-year follow-up data from some patients treated at low and high doses.
Outside the United States, uniQure plans to submit an application to the United Kingdom’s Medicines and Healthcare products Regulatory Agency, or MHRA, on the same timeline as the FDA submission.
The coming months could be a defining period for uniQure and the Huntington’s disease field. With potential license submissions, anticipated four-year data, and likely advisory committee scrutiny, AMT-130 is moving toward a critical regulatory stage as a potential first disease-modifying gene therapy for Huntington’s disease.
