uniQure Submits FDA BLA Seeking Accelerated Approval for AMT-130 Gene Therapy in Huntington's Disease
On September 2, 2026, uniQure announced the formal submission of a Biologics License Application (BLA) to the U.S. FDA seeking accelerated approval for ifezuntirgene inilparvovec (AMT-130), its investigational one-time gene therapy for Huntington’s disease. Simultaneously, the company submitted a Marketing Authorisation Application (MAA) to the United Kingdom’s Medicines and Healthcare products Regulatory Agency (MHRA).
If approved, AMT-130 would become the first-ever genetic treatment for Huntington's disease, a devastating, inherited neurodegenerative disorder. The regulatory filings are supported by three-year data from Phase I/II clinical trials demonstrating that AMT-130 can significantly slow signs of disease progression with a manageable safety profile.
The submission represents a major turnaround following regulatory hurdles. In November 2025, the FDA had initially rescinded its agreement that Phase I/II data could support a BLA. However, after further alignment and review of long-term data, the FDA agreed in June 2026 that the three-year clinical data was indeed sufficient to support a filing under the accelerated approval pathway.12 UniQure has requested a priority review, which could lead to an FDA decision within approximately eight months if accepted.
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An instance of Accelerated pathways shift the proof of clinical benefit from pre-market trials to post-marketing requirements. — The FDA accepted uniQure's BLA for accelerated approval based on early clinical results on the condition that definitive proof of efficacy will be established in post-marketing confirmatory studies. ↩︎
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An instance of Rare disease pipelines only accelerate through early FDA alignment and breakthrough designations. — Securing early alignment with the FDA allowed a rare neurodegenerative gene therapy developer to overcome prior regulatory hurdles and submit its BLA. ↩︎