April 30, 2026 —
uniQure is advancing its investigational AAV gene therapy AMT-130 for Huntington’s disease through a new regulatory pathway, following a key setback with the U.S. Food and Drug Administration.
The company plans to submit AMT-130 to the Medicines and Healthcare products Regulatory Agency (MHRA) in Q3 2026, supported by Phase 1/2 clinical data showing a 75% slowing of disease progression over three years. These data, generated from ongoing trials in the U.S. and Europe, position AMT-130 as a potentially transformative therapy for a disease with limited treatment options.
Huntington’s disease is a progressive and fatal neurodegenerative disorder caused by a mutation in the huntingtin gene, leading to motor dysfunction, cognitive decline, and psychiatric symptoms. Current therapies are largely symptomatic, underscoring the need for disease-modifying approaches.
AMT-130 utilizes an Adeno-associated virus (AAV) vector to deliver genetic material directly into the brain, with the goal of reducing production of the toxic mutant huntingtin protein. Designed as a one-time treatment, the therapy aims to provide long-term benefit following a single administration.
Despite earlier alignment with uniQure on trial design and statistical analysis, the FDA later determined that the Phase 1/2 data were insufficient to support a biologics license application (BLA). The agency is now requiring a sham surgery–controlled Phase 3 study, introducing additional complexity and timeline considerations.
In contrast, the MHRA has provided constructive feedback that supports moving forward with a submission based on existing clinical data. This divergence highlights how regulatory expectations for gene therapies—particularly in vivo AAV-based approaches—can vary significantly across regions.
uniQure continues to engage with the FDA and has scheduled a Type B meeting in Q2 2026 to discuss potential Phase 3 trial design and the role of upcoming four-year (48-month) data, expected later this year. Some experts believe these longer-term data could still offer a pathway to U.S. approval if outcomes remain favorable.
The AMT-130 program reflects broader trends in gene therapy, where durability, long-term follow-up, and clinical endpoints are becoming increasingly critical for regulatory success. At the same time, companies are adopting more flexible global strategies to navigate evolving approval landscapes.
If successful, AMT-130 could become one of the first disease-modifying gene therapies for Huntington’s disease, marking a significant milestone for both patients and the field.
