uniQure Submits BLA Seeking Accelerated FDA Approval for Huntington's Disease Gene Therapy AMT-130
On September 2, 2026, uniQure N.V. (NASDAQ: QURE) announced a major regulatory milestone with the formal submission of its Biologics License Application (BLA) to the FDA, seeking accelerated approval for ifezuntirgene inilparvovec (AMT-130). If approved, AMT-130 would become the first-ever disease-modifying genetic treatment designed to address the underlying cause of Huntington's disease. Concurrently, uniQure submitted a Marketing Authorisation Application (MAA) to the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA).
Regulatory Pathway and Breakthrough Status
The FDA has granted AMT-130 several key designations, including Breakthrough Therapy and Regenerative Medicine Advanced Therapy (RMAT), making it the first investigational Huntington's therapy to hold these dual designations. It also holds Fast Track status.
The company has formally requested Priority Review for the BLA. If accepted by the FDA, this designation would compress the agency's review cycle to six months following the standard 60-day filing review window, potentially positioning a regulatory decision in the first half of 2027.
Clinical Data and Innovative Delivery
The BLA and MAA submissions are anchored on three-year clinical data from the ongoing Phase I/II trial comparing treated patients to a propensity score-matched external control derived from the Enroll-HD natural history database. The key findings include:
- Efficacy: A single dose of AMT-130 demonstrated a statistically significant and durable slowing of Huntington's disease progression over three years.
- Mechanism of Action: AMT-130 uses a proprietary gene-silencing platform (miQURE®) incorporating a microRNA designed to silence both the mutant huntingtin gene and its highly toxic exon 1 protein fragment.
- Neurosurgical Delivery: The gene therapy is delivered via a single, MRI-guided, convection-enhanced stereotactic neurosurgical administration directly into the caudate and putamen (striatum).
uniQure plans to present a highly anticipated four-year clinical data analysis from the ongoing Phase I/II trials before the end of the third quarter of 2026, which will provide further long-term evidence of the therapy's durability and safety.