FDA Schedules Type A Meeting with uniQure to Discuss Accelerated Approval Pathway for AMT-130

January 9, 2026-The U.S. Food and Drug Administration (FDA) has scheduled a Type A meeting with uniQure to discuss materials supporting a potential accelerated approval pathway for AMT-130, the company’s investigational AAV-based gene therapy for Huntington’s disease. The meeting reopens regulatory engagement following a setback in late 2025 related to the agency’s expectations for clinical evidence.

In an announcement on January 9, 2026, uniQure stated that the meeting will focus on data supporting accelerated approval of AMT-130, an adeno-associated virus (AAV) gene therapy designed to lower mutant huntingtin protein expression. The company indicated that a further update will be provided after receipt of official FDA meeting minutes.

The scheduled discussion follows a November 2025 regulatory update in which uniQure disclosed that the FDA no longer agreed that data from ongoing Phase 1/2 AAV gene therapy trials, when compared with external natural history controls, would be sufficient to support a Biologics License Application (BLA). This position represented a change from earlier regulatory interactions, despite prior alignment on elements of an accelerated approval strategy for the AAV gene therapy program.

AMT-130 is a one-time, stereotactically delivered AAV gene therapy targeting Huntington’s disease, a fatal, inherited neurodegenerative disorder caused by a CAG repeat expansion in the HTT gene. The disease is characterized by progressive motor dysfunction, cognitive decline, and psychiatric symptoms, and currently has no approved disease-modifying treatments.

In September 2025, uniQure reported positive top-line data from its pivotal Phase 1/2 study of AMT-130. According to the company, the high-dose AAV gene therapy arm met its prespecified primary endpoint, demonstrating a statistically significant 75% slowing of disease progression at 36 months as measured by the composite Unified Huntington’s Disease Rating Scale, compared with a propensity score–matched external control cohort.

The study also achieved a key secondary endpoint, showing a 60% slowing of disease progression on total functional capacity at 36 months. AMT-130 was generally well tolerated, with a manageable safety profile and no new drug-related serious adverse events reported since December 2022. Exploratory endpoints, including cognitive and motor assessments such as the Symbol Digit Modalities Test and Stroop Word Reading Test, showed favorable trends, while cerebrospinal fluid neurofilament light levels, a biomarker of neurodegeneration, remained below baseline at 36 months.

The AMT-130 AAV gene therapy program has previously received Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT) designations, highlighting its potential to address a profound unmet medical need in Huntington’s disease. The upcoming FDA Type A meeting is expected to clarify regulatory expectations for accelerated approval of this AAV-based gene therapy approach.