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 N.V. announced the formal submission of its Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for ifezuntirgene inilparvovec (AMT-130), an investigational single-administration gene therapy for the treatment of Huntington’s disease. Simultaneously, uniQure submitted a Marketing Authorisation Application (MAA) to the United Kingdom's Medicines and Healthcare products Regulatory Agency (MHRA).

If accepted and granted priority review, a regulatory decision from the FDA could arrive as early as the second quarter of 2027. AMT-130 represents a historic milestone as the first-ever gene therapy candidate for Huntington's disease to reach the BLA submission stage.

Product Profile and Mechanism

AMT-130 is designed to be a one-time, disease-modifying treatment. It utilizes uniQure's proprietary, gene-silencing miQURE® platform to deliver a microRNA (miRNA) via an adeno-associated virus (AAV5) vector. The therapy is administered directly into the striatum (via bilateral stereotactic neurosurgery) and is specifically designed to:

1. Silence the huntingtin (HTT) gene.
2. Inhibit the production of the potentially highly toxic exon 1 protein fragment.

Clinical Evidence Supporting the BLA

The BLA and MAA submissions are supported by a comprehensive three-year clinical data analysis from the company's Phase I/II clinical trial¹ of AMT-130.

• Slowing of Disease Progression: The three-year follow-up data demonstrated a statistically and clinically meaningful slowing of disease progression in patients treated with AMT-130.
• Biomarker Evidence: Treated patients showed sustained reductions in neurofilament light chain (NfL) in cerebrospinal fluid, a key biomarker of active neuronal damage.
• Regulatory Milestones: AMT-130 has received multiple special designations from the FDA, including Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), Fast Track, and Orphan Drug designations. It also holds Advanced Therapy Medicinal Product (ATMP) classification from the European Medicines Agency (EMA).

Regulatory Context and Reversals

The submission marks a dramatic triumph for uniQure following a series of regulatory hurdles. In early 2026, the FDA had initially signaled that the Phase I/II package would be insufficient for a BLA submission, prompting intense public speculation and investor concern. However, following a productive Type B meeting and a subsequent formal reversal in mid-2026, the FDA agreed that the three-year clinical data package from the Phase I/II study was acceptable to support an accelerated approval pathway.

With the BLA officially filed, uniQure has requested a priority review from the FDA, which would compress the agency's review timeline from ten months to approximately eight months.