Following a recent U-turn from the US Food and Drug Administration (FDA), the regulatory path ahead for UniQure’s Huntington’s disease gene therapy has gained much clarity, though the biotech will likely need to navigate an advisory committee (AdCom) meeting.
“Our expectation is that we most likely will have one. We welcome it, and we are preparing for it,” said uniQure’s chief medical officer Walid Abi-Saab on a quarterly earnings call.
Go deeper with GlobalData
Discover B2B Marketing That Performs
Combine business intelligence and editorial excellence to reach engaged professionals across 36 leading media platforms.
If approved, uniQure’s gene therapy, dubbed AMT-130, would be the first therapy indicated for Huntington’s – a neurodegenerative disorder. Approximately 75,000 people in the US, Europe, and the UK have the inherited disease, which is fatal within 15 to 20 years of symptom onset. The FDA often utilises AdComs when evaluating the first drug of its kind for a specific disease or when datasets are particularly novel.
uniQure’s CEO Matthew Kapusta said: “This would be the first disease-modifying treatment for Huntington’s disease. We continue to feel like our interactions with the FDA have been constructive and productive. Our view is that the data speaks for itself and we would very much look forward to [in the event of] an AdCom.”
UniQure’s AMT-130 has already had a less than straightforward regulatory journey. The agency refused to accept the biotech’s filing for AMT-130 in November 2024, citing insufficient data from a Phase I/II trial. After meeting with the FDA in January, uniQure relayed that the FDA was demanding another study.
Following another meeting in June, the FDA changed its tune. While it is unclear what prompted the decision, the agency said the three-year analysis from the Phase I/II study is an acceptable data package that can form a Biologics Licence Application (BLA) for the accelerated approval of AMT-130.
The FDA and uniQure still need to align on the design of a confirmatory study prior to the BLA submission. As per new guidance for accelerated approvals, the confirmatory study should be underway, and ideally fully enrolled, by the time the FDA grants the expedited marketing authorisation. For its part, uniQure said it will begin the study as quickly as possible once aligned with the FDA.
In September 2026, uniQure is planning to present data from its ongoing Phase I/II studies of AMT-130. The update is expected to include four-year follow-up data from some of the patients treated with AMT-130 at low and high doses.
Outside the US, uniQure has confirmed it plans to submit an application to the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) on the same timeline as the FDA.
Kapusta added: “We entered the second half of 2026 with clarity on a regulatory pathway for AMT-130, both in the US and UK. With the submission of multiple license applications for AMT-130 and the anticipated release of four-year data, the coming months represent a potentially transformational milestone for uniQure and for the Huntington’s disease community we are committed to serving.”
uniQure, which currently only has one approved product in the form of haemophilia B gene therapy Hemgenix, reported Q2 revenue of $5.8m. As of June’s end, the biotech had $810.3m in cash, enough to support activities into 2030.
Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva.
Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.
