uniQure Prepares for Potential FDA Panel on Huntington’s Gene Therapy After Priority Review Nod

Key Points

• FDA accepted uniQure’s AMT-130 application for priority review, and the company is preparing for a potential advisory committee meeting. The review does not guarantee approval.
• The application relies on 36- and 48-month results compared with an external natural-history control; CEO Matt Kapusta cited better preservation of daily functioning among treated patients, while acknowledging that more than half of control data points were missing at 48 months.
• If AMT-130 receives accelerated approval, uniQure plans a roughly 200-patient confirmatory study. The company is also preparing for a potential launch and said its approximately $780 million in cash is expected to fund operations into 2030.

uniQure NASDAQ: QURE is preparing for a potential U.S. advisory committee meeting on AMT-130, its experimental gene therapy for Huntington’s disease, after regulators accepted its biologics license application for priority review, CEO Matt Kapusta said.

Kapusta said a potential advisory committee meeting was in line with the company’s expectations for a one-time gene therapy seeking to become the first disease-modifying treatment for Huntington’s. He said uniQure began preparing months ago and has engaged an outside adviser and planned mock panels.

“We’re delighted to have an opportunity to speak about our data in public and to have perspectives from various stakeholders, including the patient community,” Kapusta said. He declined to predict what questions the Food and Drug Administration might raise.

FDA Review Follows Shift in Feedback

Kapusta described a change in the FDA’s feedback earlier in 2026. At a January meeting, he said, the agency told uniQure that the 36-month data presented to date could not serve as the primary analysis for a license application and strongly recommended a double-blinded, sham-controlled Phase 3 study.

At a June meeting that included FDA leadership, the agency said the data could support the primary basis of an application, according to Kapusta. He declined to speculate on what prompted the change, but said the company had continued preparing its submission.

The application is based on data from September 2025 and follows a statistical analysis plan previously shared with the FDA, Kapusta said. The company has used natural history data to create an external control group for comparison with treated patients.

Kapusta said uniQure tested assumptions underlying that comparison, including using another natural history dataset and different patient-matching characteristics. The main source, Enroll-HD, had more than 30,000 participants when the company conducted its original analysis in 2025, he said.

Four-Year Data and Control Group Limitations

Kapusta said more recent data presented in September supported the application’s primary analysis. At 48 months, treated patients had lost, on average, less than one-tenth of a point on Total Functional Capacity, a measure of independence in daily activities, compared with nearly one point for untreated patients, he said.

He also pointed to a difference between high- and low-dose patients on a composite Huntington’s disease rating scale as evidence consistent with a dose-dependent treatment effect.

Kapusta cautioned that the external control became more limited over time. More than half of its data points were missing at 48 months, he said, and patients who discontinued appeared to have been progressing more rapidly than those who remained. That left a comparatively healthier control population at four years, he said.

“We believe the drug is approvable, and that we stand by the data,” Kapusta said. FDA acceptance of an application and priority review do not guarantee approval.

Confirmatory Study and Launch Planning

For a confirmatory study intended to support full approval if AMT-130 receives accelerated approval, uniQure expects to enroll about 200 patients, Kapusta said. Patients will be randomized to treatment or standard of care, with their progress compared at 36 months using Total Functional Capacity. The study will not be blinded to patients or providers and will not include a sham procedure, he said.

Kapusta did not provide an enrollment target for the May 1 review date discussed during the event. He said the company expects to have multiple sites activated and screening patients, as well as patients enrolled, by then.

Although labeling discussions have not begun, uniQure is preparing for a potential launch. Kapusta said administration requires precision surgical delivery using a specific catheter system, and the company expects a minimum of 15 sites to be available for the procedure. A separate U.K. license application has been validated and is under review, he added.

Beyond Huntington’s disease, uniQure expects additional data from its temporal lobe epilepsy and Fabry disease programs in the first half of 2027. Kapusta said the company has approximately $780 million in cash, which it expects to fund operations into 2030.

About uniQure (NASDAQ:QURE)

uniQure N.V. is a biotechnology company that develops gene therapies for serious genetic and other diseases. The company uses adeno-associated virus (AAV) vectors to deliver genetic material to patients' cells, with research and development operations in the United States and the Netherlands. Its programs have targeted conditions including Huntington's disease, epilepsy and hemophilia B. uniQure developed the gene therapy now known as Hemgenix for hemophilia B; it is commercialized by CSL Behring under a partnership.

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