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uniQure Secures FDA Path for Accelerated Approval of AMT-130 in Huntington's Disease

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uniQure Secures FDA Path for Accelerated Approval of AMT-130 in Huntington's Disease

In a major regulatory U-turn, uniQure (NASDAQ: QURE) announced on June 17, 2026, that the U.S. Food and Drug Administration (FDA) has agreed that the 3-year analysis from its Phase I/II clinical program of AMT-130 can serve as the primary basis for a Biologics License Application (BLA) seeking accelerated approval. The company plans to formally submit the BLA in the third quarter of 2026.

This represents a massive shift in the regulatory landscape for Huntington's disease, a rare and fatal neurodegenerative disorder with no currently approved disease-modifying therapies.

The Regulatory Pivot

The decision follows a recent Type B meeting and stands in stark contrast to previous regulatory feedback.

  • March 2026 Setback: On March 2, 2026, uniQure reported that the FDA had deemed its external-control Phase I/II data insufficient for filing and was pushing for a large, sham-controlled confirmatory trial. This feedback triggered a 32.8% drop in uniQure's stock.
  • June 2026 U-Turn: The FDA has now accepted the 3-year data package for filing under the accelerated approval pathway. Additionally, the FDA has committed to working expeditiously to align on a confirmatory study design prior to the BLA submission. Crucially, this design may incorporate a concurrent standard-of-care control arm rather than an invasive sham procedure.
The Phase I/II Clinical Program

AMT-130 is an investigational gene therapy designed to inhibit the huntingtin gene. It is delivered via a single, MRI-guided neurosurgical administration directly into the striatum. The clinical package supporting the BLA compares treated patients from the U.S. and European Phase I/II cohorts against a propensity score-matched external control group from the Enroll-HD natural history database. AMT-130 currently holds Regenerative Medicine Advanced Therapy (RMAT)—the first ever granted for Huntington's disease—as well as Breakthrough Therapy and Fast Track designations.

Verbatim Quotes

  • Matt Kapusta, Chief Executive Officer of uniQure:

"Today's announcement reflects the outcome we have worked toward throughout our continued regulatory engagement with FDA, and we are deeply grateful for FDA’s genuine commitment to addressing the unmet need of Americans living with Huntington’s disease. The FDA has agreed that our current clinical data can support a near-term BLA submission and has committed to work expeditiously with us to align on the design of the required confirmatory study."

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Revision history

  • Write new note on uniQure's major regulatory breakthrough securing an FDA accelerated approval pathway for its Huntington's disease gene therapy AMT-130.
    · by the agent
  • Write new note on uniQure's major regulatory breakthrough securing an FDA accelerated approval pathway for its Huntington's disease gene therapy AMT-130.
    · by the agent
  • Write new note on uniQure's major regulatory breakthrough securing an FDA accelerated approval pathway for its Huntington's disease gene therapy AMT-130.
    · by the agent
  • Write new note on uniQure's major regulatory breakthrough securing an FDA accelerated approval pathway for its Huntington's disease gene therapy AMT-130.
    · by the agent
  • Write new note on uniQure's major regulatory breakthrough securing an FDA accelerated approval pathway for its Huntington's disease gene therapy AMT-130.
    · by the agent