uniQure Nears BLA Submission for AAV-Based Gene Therapy AMT-130 for Huntington’s Disease

LEXINGTON, Mass. & AMSTERDAM – June 2, 2025 – uniQure today announced significant progress in its regulatory discussions with the U.S. Food and Drug Administration (FDA) for AMT-130, its investigational AAV-based gene therapy designed to treat Huntington’s disease. The company has reached alignment with the FDA on critical aspects of the Biologics License Application (BLA) submission, which is now anticipated in the first quarter of 2026.

This pivotal update follows recent Type B meetings with the FDA, where key components of the statistical analysis plan (SAP) and Chemistry, Manufacturing and Controls (CMC) information for this AAV gene therapy were finalized. The collaboration with the FDA underscores the accelerated approval pathway uniQure is pursuing for AMT-130, a move that reflects the urgent need for disease-modifying treatments in Huntington’s disease.

“We are very pleased with our continued, productive engagement with the FDA and the progress we’ve made toward a planned BLA submission for AMT-130 in the first quarter of 2026,” stated Walid Abi-Saab, M.D., chief medical officer of uniQure. “Our rigorous and differentiated approach, supported by multiple years of clinical data, is geared towards delivering the first disease-modifying treatment for people affected by this devastating disease, leveraging the precision of AAV gene delivery.”

Key Alignments with FDA:

• Statistical Analysis Plan: The FDA affirmed that the composite Unified Huntington’s Disease Rating Scale (cUHDRS) is an acceptable intermediate clinical endpoint for accelerated approval. The primary efficacy analysis for the BLA will compare the 3-year change in cUHDRS in high-dose AMT-130 patients to a propensity score-adjusted external control arm derived from the extensive ENROLL-HD dataset. This significantly larger dataset, compared to previous natural history studies, is expected to enhance the robustness of the analysis for this AAV-mediated therapy. uniQure plans to submit an updated SAP to the FDA this quarter.
• CMC Requirements: The FDA concurred that the validation of AMT-130’s AAV manufacturing process can leverage prior knowledge and experience from the company’s approved AAV gene therapy, HEMGENIX, complemented by additional full-scale GMP batches and a single Process Performance Qualification (PPQ) batch. The proposed drug product release testing plan for this AAV therapeutic, including the potency assay, also received FDA agreement, pending final qualification.

AMT-130 has previously received the FDA’s Regenerative Medicine Advance Therapy (RMAT) designation and Breakthrough Therapy designation, highlighting its potential as a significant therapeutic advancement for Huntington’s disease, a rare inherited neurodegenerative disorder with no approved disease-modifying treatments.

uniQure is conducting two multi-center, dose-escalating, Phase I/II clinical studies of AMT-130 in the U.S. and Europe, exploring its safety, tolerability, and efficacy signals. The studies involve a single administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum, demonstrating the targeted delivery approach of this AAV gene therapy.

About Huntington’s Disease: Huntington’s disease is a devastating inherited neurodegenerative disorder characterized by motor symptoms, behavioral abnormalities, and cognitive decline. It is caused by a genetic mutation leading to the production of abnormal protein in the brain, with approximately 70,000 diagnosed patients in the U.S. and Europe. Currently, there are no approved therapies to delay or slow the progression of this severe disease, underscoring the potential of AAV gene therapies like AMT-130.