uniQure (QURE) Updates AMT-130 FDA Feedback; BLA Timing Uncertain
On November 3, 2025, uniQure N.V. (NASDAQ: QURE) announced feedback from the U.S. Food and Drug Administration (FDA) regarding AMT-130, its investigational gene therapy aimed at treating Huntington’s disease. This feedback was delivered during a pre-Biologics License Application (BLA) meeting.
FDA Feedback on AMT-130 and Impact on BLA Timing
uniQure reported that the FDA now disagrees with the adequacy of Phase I/II data against an external control as primary evidence for the BLA submission. This represents a significant shift from prior Type B meeting guidance, leaving the timing of the BLA submission uncertain.
Future Steps and Agency Engagement
- uniQure anticipates receiving final meeting minutes within 30 days.
- The company plans urgent discussions with the FDA to clarify the next steps.
- Parallel regulatory discussions will also be held with agencies in the European Union and the United Kingdom.
Designations and Previous Guidance
AMT-130 received Breakthrough Therapy designation in April 2025 and Regenerative Medicines Advanced Therapy (RMAT) designation in May 2024. These designations support the potential for expedited review and approval.
CEO Matt Kapusta expressed surprise and disappointment at the FDA’s recent feedback, noting its deviation from previous guidance received in November 2024, which indicated that the existing Phase I/II data could support a BLA submission.
Next Steps for AMT-130
uniQure remains committed to engaging with the FDA to explore pathways for AMT-130’s approval. The outcome of these discussions will significantly impact the regulatory trajectory for this promising gene therapy aimed at providing relief for patients suffering from Huntington’s disease.
Conclusion
The company is closely monitoring upcoming interactions with regulatory authorities. The clarity on BLA submission timing and potential requirements for additional evidence remains a priority as uniQure navigates these developments.
