uniQure Faces Unexpected FDA Setback on Huntington Disease Gene Therapy BLA

LEXINGTON, MA — November 4, 2025— uniQure, a biotechnology company developing gene therapies, announced an unexpected roadblock in the regulatory path for its adeno-associated virus (AAV) vector-based gene therapy, AMT-130, intended to treat Huntington disease (HD).

Based on feedback from a recent pre-Biologics License Application (preBLA) meeting, uniQure no longer believes the U.S. Food and Drug Administration (FDA) will find data from its Phase 1/2 studies, compared to an external control, sufficient to support a BLA submission for AMT-130.

A Drastic Change in Regulatory Guidance

The company noted this feedback represents a “drastic change” from guidance previously given by the FDA during multiple Type B meetings, including one in December 2024. At that time, the FDA had indicated alignment with uniQure’s plan to submit a BLA based primarily on Phase 1/2 data using the Accelerated Approval Pathway. The agency had previously agreed that the composite Unified Huntington’s Disease Rating Scale (cUHDRS) could serve as an intermediate clinical end point, supported by decreases in cerebrospinal fluid neurofilament light chain (NfL). “We are surprised by the FDA’s feedback at the recent preBLA meeting, which is a drastic change from the guidance the FDA provided in November 2024,” said Matt Kapusta, MBA, CEO of uniQure. “This news is unexpected, and we are truly disappointed for people living with HD, who have no disease-modifying treatment options for this devastating disease.”

The company is currently awaiting the final minutes of the preBLA meeting, expected within 30 days, but stated that the expected timeframe for a BLA submission is now unclear.

Prior Clinical Success

The unexpected regulatory shift follows promising data announced in September 2025 from one of the Phase 1/2 trials. The study reported that, at 36 months post-treatment:

• Primary Endpoint Met: High-dose AMT-130 was shown to significantly slow disease progression by 75% compared to a propensity score-matched external control, as assessed by cUHDRS.
• Key Secondary Endpoint Met: A statistically significant 60% slowing of disease progression compared to the external control was also reported via Total Functional Capacity (TFC).

“I believe these groundbreaking data are the most convincing in the field to date and underscore potential disease-modifying effects in HD,” said Dr. Sarah Tabrizi, a professor of clinical neurology and HD expert, at the time the data was released.

Despite the setback, uniQure stated its intent to continue interaction with the FDA to find a path for “timely accelerated approval” and plans to advance discussions with regulatory bodies outside the United States.