TLDR:
- FDA seeks more data before advancing uniQure’s AMT-130 gene therapy.
- uniQure plans follow-up meeting with FDA for AMT-130 approval process.
- FDA requests further engagement for AMT-130 submission, delaying approval.
- uniQure faces setback, aims for 2026 FDA meeting on AMT-130.
- AMT-130’s Huntington’s disease treatment advances after FDA feedback.
uniQure (NASDAQ: QURE) saw a significant drop in its stock price, falling 12.28% to $22.35 after the FDA’s request for additional engagement on its investigational gene therapy AMT-130.
uniQure N.V., QURE
The FDA’s decision comes after a pre-BLA (Biologics License Application) meeting on October 29, 2025, where it indicated that the available Phase I/II data may not support a BLA submission for AMT-130 at this stage. The company plans to request a follow-up meeting in early 2026 to address the feedback and move the therapy closer to approval.
FDA Seeks Additional Data for AMT-130 Submission
UniQure received feedback from the FDA, indicating that the current Phase I/II trial data might not provide the primary evidence needed for a BLA submission. The agency’s request for further engagement has delayed the progression of AMT-130 towards approval. The company is now evaluating the FDA’s feedback and plans to schedule a follow-up meeting with the agency in Q1 2026 to discuss the next steps.
This setback comes as a disappointment to uniQure, as the company had hoped the existing Phase I/II data would support the BLA submission. However, the FDA’s stance underscores the need for more detailed data before AMT-130 can advance in the regulatory process. uniQure remains committed to addressing the FDA’s concerns and continues working towards moving the therapy closer to approval.
uniQure’s Commitment to Huntington’s Disease Treatment
uniQure has reiterated its commitment to advancing AMT-130, emphasizing the critical need for new therapies for Huntington’s disease. The company has gained significant support from the Huntington’s disease community, including patients, families, and clinicians, who view AMT-130 as a potential breakthrough treatment. This backing strengthens uniQure’s resolve to continue pushing the gene therapy through the regulatory process.
UniQure’s CEO, Matt Kapusta, emphasized the importance of collaboration with the FDA in order to address the regulatory requirements. He also noted the strong community support for the therapy, which highlights the pressing need for effective treatments for Huntington’s disease. Despite the regulatory challenges, uniQure remains focused on meeting the FDA’s requirements and bringing AMT-130 closer to approval.
Path Forward for AMT-130: Overcoming Regulatory Hurdles
The regulatory journey for AMT-130 is ongoing, and uniQure’s request for a follow-up meeting with the FDA is a crucial part of its strategy. The company remains committed to obtaining the necessary data to support a future BLA submission and hopes to resolve the agency’s concerns. As AMT-130 continues to show promise in early trials, uniQure remains determined to overcome the hurdles in the regulatory process and bring a much-needed treatment to patients.
UniQure’s next steps will play a pivotal role in determining AMT-130’s future development. The company is focused on ensuring the therapy meets the necessary standards for approval. With ongoing efforts to navigate the regulatory landscape, uniQure aims to move AMT-130 closer to becoming a viable treatment for Huntington’s disease.
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