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FDA Accepts uniQure Huntington Gene Therapy Data, 75% Progression Cut

6/18/2026

Summary

uniQure Gains FDA

The FDA agreed to accept Phase 1/2 data for approval of uniQure’s AMT-130 gene therapy for Huntington’s disease.
The trial showed a 75% reduction in disease progression for high-dose patients after three years.
uniQure will file a license application in Q3 2026, with shares up 78% to $49.49 pre-market.
After commissioner Marty Makary and director Vinay Prasad left, FDA leadership works with uniQure on a study using a standard control, not sham surgery.

Summary

uniQure Files UK

uniQure will file a marketing authorization for AMT-130 with the UK Medicines and Healthcare products Regulatory Agency (MHRA) in Q3 2026 after a pre-submission meeting.
The MHRA gave constructive feedback, unlike the FDA’s earlier demand for a Phase 3 trial.
AMT-130 holds Regenerative Medicine Advanced Therapy, Breakthrough Therapy and Fast Track designations, a rare “triple crown.”
The FDA now prefers a concurrent standard-of-care control for the confirmatory study, dropping the sham-surgery arm.

History

Historical Context

In November 2012 the European Commission granted UniQure’s Glybera the EU’s first gene-therapy marketing authorisation, pricing the single dose at about €1 million and treating only 31 patients before withdrawal in 2017. That breakthrough showed regulators would accept high-cost, rare-disease therapies and flexible trial designs, a model UniQure now cites as it pursues FDA and MHRA approval for its AAV-based neurodegenerative candidate AMT-130.

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uniQure Gains FDA

The FDA agreed to accept Phase 1/2 data for approval of uniQure’s AMT-130 gene therapy for Huntington’s disease.
The trial showed a 75% reduction in disease progression for high-dose patients after three years.
uniQure will file a license application in Q3 2026, with shares up 78% to $49.49 pre-market.
After commissioner Marty Makary and director Vinay Prasad left, FDA leadership works with uniQure on a study using a standard control, not sham surgery.

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