uniQure N.V. (QURE) said it has completed a successful pre-submission meeting with the UK Medicines and Healthcare products Regulatory Agency (MHRA), clearing the path for a planned Marketing Authorization Application (MAA) for AMT-130 in Huntington's disease in the third quarter of 2026.
The company described the interaction as "constructive," noting that regulators reviewed the data package and manufacturing requirements needed to support the submission.
Huntington's disease is a rare, inherited neurodegenerative disorder that progressively impairs movement, cognition, and behavior. AMT-130, uniQure's investigational gene therapy, is being evaluated in ongoing U.S. and European Phase I/II trials. According to the company, the three-year data showed a statistically significant 75% slowing of disease progression at the high dose compared with a matched external control group, with a generally manageable safety profile.
The MHRA meeting focused on the clinical dataset and manufacturing readiness required for an MAA. uniQure said it expects to submit the application based on the three-year analysis from both Phase I/II studies. In parallel, the company has secured a Type B meeting with the U.S. FDA in the second quarter of 2026 to discuss the potential Phase III trial design and the statistical analysis plan for the upcoming four-year data readout expected in the third quarter of 2026.
uniQure also noted it is actively pursuing additional regulatory pathways outside the U.S. and UK to support potential global registration of AMT-130, with further updates expected in the second half of 2026.
QURE has traded between $8.73 and $71.50 over the past year. The stock is currently trading at $19.12, up 14.20%.
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