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uniQure Provides Regulatory Update on AMT-130 for Huntington's Disease

uniQure N.V. (QURE) | December 4, 2025

By Bob Smith

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uniQure received final meeting minutes from the FDA regarding a pre-BLA meeting for AMT-130

FDA conveyed that data from Phase I/II studies of AMT-130 may not support BLA submission

uniQure plans to request a follow-up meeting with the FDA in Q1 2026

FDA Feedback

Data from Phase I/II studies may not support BLA submission

Community Support

Strong support from Huntington's disease community for urgent treatment advancement

Company Commitment

uniQure committed to collaborating with FDA for patient access to AMT-130

  • The FDA's feedback indicates potential challenges in the BLA submission process for AMT-130
  • Strong community support highlights the urgent need for effective treatments in Huntington's disease

Despite challenges, uniQure remains dedicated to advancing AMT-130 in collaboration with the FDA and the Huntington's disease community.