uniQure Targets Q3 UK Marketing Authorization Submission for Huntington’s Gene Therapy
Event summary
- uniQure plans to submit a UK Marketing Authorization Application for AMT-130 in Q3 2026, following constructive feedback from the MHRA.
- Three-year Phase I/II trial data showed a 75% slowing of disease progression (p=0.003) and a manageable safety profile at high dose.
- Type B meeting with FDA scheduled for Q2 2026 to discuss potential Phase III design and four-year analysis plan.
- uniQure is pursuing additional ex-US regulatory pathways for AMT-130.
The big picture
uniQure’s progress with AMT-130 reflects growing regulatory openness to gene therapies for rare neurodegenerative diseases. The UK’s MHRA has emerged as a faster alternative to the FDA for some biotech approvals, while uniQure’s parallel US strategy highlights the complexity of global commercialization. Success here could validate its pipeline beyond hemophilia B.
What we're watching
- Regulatory Momentum
- Whether uniQure can secure parallel approval pathways in the US and UK by 2027.
- Clinical Validation
- How four-year Phase I/II data will impact FDA’s view of AMT-130’s efficacy.
- Market Expansion
- The pace at which uniQure advances ex-US registrations for Huntington’s therapy.
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