uniQure to File Q3 2026 AMT-130 UK MAA After 75% Huntington’s Slowing
QURE•uniQure expects to submit a UK MAA for AMT-130 in Q3 2026 following an MHRA pre-submission meeting that reviewed its three-year Phase I/II results showing a 75% disease progression slowdown (p=0.003). A Type B FDA meeting in Q2 2026 will discuss Phase III design and four-year data.
1. MHRA Pre-Submission Meeting Outcomes
uniQure held a Type I pre-submission meeting with the UK MHRA to review its AMT-130 dossier and manufacturing requirements. The feedback was constructive, allowing the company to plan a Marketing Authorization Application submission in the third quarter of 2026 based on a three-year clinical data package.
2. Three-Year Phase I/II Efficacy Data
Data from ongoing U.S. and European Phase I/II trials showed a statistically significant 75% slowing of Huntington’s disease progression at the high dose versus an external control (p=0.003). The therapy was generally well tolerated with a manageable safety profile across the cohort.
3. Upcoming FDA and Global Regulatory Plans
uniQure secured a Type B meeting with the FDA in Q2 2026 to align on Phase III trial design and a statistical analysis plan for the four-year dataset expected in Q3 2026. The company is also pursuing additional ex-US regulatory pathways to support potential international registrations.
