uniQure Announces Agreement with FDA on Key Aspects of Accelerated Approval Pathway for AMT-130 in Huntington's Disease

uniQure , has reached an important agreement with the U.S. Food and Drug Administration (FDA) on key components of an Accelerated Approval pathway for AMT-130 in Huntington’s disease. This development brings AMT-130 closer to offering a potential life-changing therapy to patients suffering from this devastating neurodegenerative disorder.

A Major Milestone for Huntington’s Disease Treatment

AMT-130, a gene therapy designed to target the root cause of Huntington’s disease, has been under investigation in ongoing Phase I/II clinical trials. The FDA's agreement on key elements, including the use of data from these studies and the composite Unified Huntington’s Disease Rating Scale (cUHDRS) as an intermediate clinical endpoint, paves the way for a Biologics License Application (BLA) under the Accelerated Approval pathway. This is a crucial step in the development of AMT-130 as it aims to address the unmet medical needs of Huntington’s disease patients.

Key FDA Agreements

During a Regenerative Medicine Advanced Therapy (RMAT) Type B meeting held in November 2024, the FDA confirmed that:

Ongoing Phase I/II study data, compared to a natural history external control, may serve as the primary basis for the BLA submission.

The cUHDRS scale will be used as an intermediate clinical endpoint for Accelerated Approval.

Reductions in neurofilament light chain (NfL), measured in cerebrospinal fluid (CSF), will serve as supportive evidence of therapeutic benefit.

A Groundbreaking Therapy for Huntington’s Disease

AMT-130 is designed to provide a long-term solution by addressing the root cause of Huntington’s disease mutant huntingtin protein aggregation through a single administration. Preliminary clinical data from the VISION-HD study has shown durable, dose-dependent slowing of disease progression and reductions in CSF NfL, a marker of neurodegeneration.

A Bright Future for Huntington’s Disease Patients

With RMAT designation granted to AMT-130 in May 2024, uniQure is progressing toward BLA readiness. The company is working closely with the FDA and expects to further discuss the statistical analysis plan and technical requirements in the first half of 2025.

AMT-130 holds promise to transform the treatment landscape for Huntington’s disease, offering patients a potential disease-modifying therapy. Huntington’s disease, a rare inherited neurodegenerative disorder, currently lacks approved therapies to slow its progression, and AMT-130 could offer a crucial solution for patients in need.

About the Phase I/II Clinical Program of AMT-130

uniQure’s Phase I/II clinical studies for AMT-130 are multi-center, dose-escalating trials investigating the safety, tolerability, and efficacy of this gene therapy. The studies have been conducted in the U.S. and Europe, with patients receiving a single administration of AMT-130 via MRI-guided surgery. The treatment aims to halt or slow disease progression by reducing the accumulation of abnormal huntingtin protein in the brain.

Looking Ahead

As uniQure prepares for BLA submission and continued collaboration with the FDA, the company remains dedicated to bringing this potential life-changing therapy to Huntington’s disease patients worldwide. This milestone is a critical step forward for the Huntington’s disease community, and AMT-130 represents a beacon of hope for those living with this debilitating condition.