uniQure Submits BLA to FDA and MAA to UK MHRA for Huntington's Gene Therapy AMT-130
QURE has more than doubled off its 52-week low of $8.73 on light trading volume (0.4× avg).
Summary
uniQure submitted its BLA to the FDA and MAA to the UK MHRA for AMT-130, its Huntington's disease gene therapy, and requested priority review. The submissions are supported by three-year Phase I/II data showing slowed disease progression.
Key Events · Product Development and Regulatory · QURE
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BLA Submitted to FDA
uniQure submitted a Biologics License Application to the FDA for accelerated approval of AMT-130 for Huntington's disease, supported by three-year Phase I/II data showing slowed disease progression.
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MAA Submitted to UK MHRA
A Marketing Authorisation Application for AMT-130 was also submitted to the UK's MHRA, expanding the regulatory pathway into Europe.
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Priority Review Requested
The company requested priority review for the BLA, which if granted would shorten the FDA review cycle to six months following the 60-day filing review period.
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Four-Year Data Presentation Planned
uniQure intends to present a four-year data analysis from the ongoing Phase I/II studies before the end of Q3 2026.
Analysis · QURE · Life Sciences
A major regulatory milestone has been reached: uniQure has formally submitted its Biologics License Application to the FDA for accelerated approval of AMT-130 in Huntington's disease, and separately submitted a Marketing Authorisation Application to the UK MHRA. The company also requested priority review, which if granted would shorten the FDA review cycle to six months after the 60-day filing review period. This is the first gene therapy candidate for Huntington's disease to reach this stage, and the submissions are backed by three-year Phase I/II data showing slowed disease progression. The company plans to present four-year data before the end of Q3 2026. This is a major regulatory milestone that moves AMT-130 from clinical development into the approval process, with potential for accelerated approval and a near-term commercial launch.
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At the time of this filing, QURE was trading at $48.34 on NASDAQ in the Life Sciences sector, with a market capitalization of approximately $3.3B. The 52-week trading range was $8.73 to $71.50. This filing was assessed with positive market sentiment and an importance score of 8 out of 10.