uniQure is pursuing UK regulatory approval for its Huntington’s disease gene therapy AMT-130 after the FDA rejected the company’s initial approval pathway in early 2026. The biotech firm plans to submit a marketing authorization application to the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) in the third quarter of 2026, following a successful pre-submission meeting held in April.
The shift to the UK marks a strategic pivot after the FDA declined to accept uniQure’s Phase 1/2 clinical data as sufficient for approval. In November 2025, the company announced it was no longer aligned with the FDA on how to advance AMT-130, and in March 2026 the agency formally demanded a Phase 3 trial instead of approving the therapy based on earlier-stage results.
AMT-130 demonstrated a statistically significant 75% slowing of Huntington’s disease progression at 36 months in the Phase 1/2 trial, measured by the composite Unified Huntington’s Disease Rating Scale at the high dose. The therapy is a one-time gene treatment that uses an adeno-associated virus to deliver microRNA directly into the brain to lower levels of the huntingtin protein that causes the fatal neurodegenerative disorder.
uniQure CEO Matt Kapusta said the company was “encouraged by the constructive feedback from the MHRA,” signaling a more receptive stance from the UK regulator than the FDA has shown. The MHRA’s approval of the three-year data package for a marketing authorization application contrasts with the FDA’s insistence on additional trial data.
The company is also pursuing a parallel path in the United States. It has been granted a Type B meeting with the FDA scheduled for the second quarter of 2026 to discuss a potential Phase 3 trial design and statistical analysis plan for four-year data expected in the third quarter of 2026. uniQure is actively exploring additional regulatory pathways in international markets beyond the UK.
The FDA-uniQure dispute has raised broader questions about gene therapy approval standards. When the agency initially granted AMT-130 breakthrough therapy designation in April 2025 based on the Phase 1/2 data, uniQure had believed it was aligned with the FDA on an accelerated approval pathway. The reversal sparked debate within the rare disease community about regulatory consistency.
QURE stock price on June 16, 2026 closed at $26.99, down significantly from peaks earlier in the year when the company announced its clinical trial results. The stock has faced headwinds as investors weighed the FDA setback against the potential for UK and international approvals.
Sources
- uniQure investor relations — April 30, 2026 press release announcing UK MHRA pre-submission meeting and Q3 2026 submission timeline
- BioPharma Dive — April 30, 2026 report on uniQure’s UK approval strategy following FDA rejection
- BioSpace — April 30, 2026 coverage of uniQure’s pivot to UK approval after FDA disagreement
- CNBC — March 5, 2026 report on FDA official’s statement that AMT-130 requires additional study data
- Clinical Trials Arena — September 25, 2025 report on 75% disease progression slowing in AMT-130 trial
- Yahoo Finance — June 16, 2026 QURE stock price and YTD performance data
- Macrotrends — June 16, 2026 QURE closing price
