uniQure Submits Historic BLA for Huntington's Disease Gene Therapy AMT-130

uniQure Submits Historic BLA for Huntington's Disease Gene Therapy AMT-130

On September 2, 2026, uniQure formally submitted its Biologics License Application (BLA) to the U.S. FDA seeking accelerated approval¹ for ifezuntirgene inilparvovec (AMT-130), its investigational AAV5 gene therapy for Huntington's disease. Simultaneously, the company submitted a Marketing Authorisation Application (MAA) to the UK's Medicines and Healthcare products Regulatory Agency (MHRA).

If approved, AMT-130 will represent a landmark milestone as the first-ever genetic therapy and the first disease-modifying treatment for Huntington’s disease, a devastating and fatal inherited neurodegenerative disorder that currently has no approved therapies to slow disease progression.

Clinical Foundation and the FDA Reversal

The regulatory filings are supported by a three-year clinical data analysis from uniQure's ongoing Phase I/II studies in the U.S. and Europe. In these trials, a single administration of AMT-130 delivered directly into the striatum (caudate and putamen) via targeted, MRI-guided stereotactic neurosurgery demonstrated a significant slowing of disease progression:

• Efficacy: The three-year data demonstrated preservation of motor and cognitive function compared to natural history controls.
• Natural History Comparison: The submission follows a previous regulatory hurdle where the FDA initially questioned uniQure's use of an external natural history database (Enroll-HD) rather than an in-study control group to establish long-term clinical benefit. The FDA's subsequent reversal and acceptance of this comparison paved the way for the current BLA filing.

Mechanism of Action

AMT-130 utilizes uniQure's proprietary miQURE gene-silencing platform. It delivers a microRNA (miRNA) designed to target and silence the huntingtin (HTT) gene, thereby reducing the production of both mutant huntingtin protein and its highly toxic exon 1 fragment.