uniQure Shares FDA Feedback Update; BLA Timeline for AMT-130 Uncertain

uniQure N.V. (NASDAQ: QURE), a gene therapy company focused on treating severe medical conditions, encountered unexpected feedback from the U.S. Food and Drug Administration (FDA) during a pre-BLA meeting regarding its investigational gene therapy, AMT-130, developed for Huntington’s disease. This feedback, received on November 3, 2025, signifies a notable change in regulatory expectations for the progression of the therapy.

Key Updates on AMT-130 and FDA Feedback

The FDA has expressed concerns that the data from Phase I/II studies comparing AMT-130 to an external control may not suffice as primary evidence for a Biologics License Application (BLA). This perspective contrasts significantly with previous guidance provided during multiple Type B meetings over the past year, where the FDA indicated that such data could potentially support a BLA submission.

Timeline and Next Steps

As a consequence of this feedback, the timeline for the BLA submission has become unclear. uniQure anticipates obtaining the final meeting minutes within approximately 30 days. In response, the company plans to engage urgently with the FDA to explore alternative pathways for expedited approval.

• Feedback Date: November 3, 2025
• Designation Status:
  • Breakthrough Therapy: Granted in April 2025
  • Regenerative Medicines Advanced Therapy (RMAT): Granted in May 2024

Ongoing Regulatory Engagement

Besides its communication with the FDA, uniQure aims to initiate discussions with regulatory bodies in the European Union and the United Kingdom, ensuring a comprehensive approach to advancing AMT-130.

The CEO of uniQure, Matt Kapusta, expressed surprise and disappointment regarding the FDA’s recent stance. He emphasized the necessity of continuing efforts to validate the therapy’s potential benefits for Huntington’s disease patients, who currently lack effective disease-modifying options.

Future Prospects for AMT-130

The shifting regulatory landscape introduces uncertainties related to the approval process. The reliance on external-control data as primary evidence now poses challenges for AMT-130’s expedited approval trajectory. uniQure remains committed to collaborating with the FDA to define a viable path forward relating to its ongoing clinical trials and regulatory submissions.

Status updates concerning the final meeting minutes and outcomes of urgent FDA engagements will be critical in determining the near-term regulatory pathway for AMT-130. The company continues to navigate the complexities inherent in bringing this potentially transformative therapy to market.