uniQure N.V. (QURE) Stock: Plunges 57% After Unexpected FDA Setback on AMT-130

TLDR

• uniQure’s stock plummets 57% after an unexpected FDA setback on AMT-130.
• FDA throws a wrench in uniQure’s AMT-130 approval, causing a 57% stock drop.
• uniQure’s AMT-130 faces delay after FDA questions data adequacy, stock dives.
• uniQure’s stock crashes as FDA challenges AMT-130’s approval pathway.
• Stock falls 57% as FDA feedback forces uniQure to rethink AMT-130 approval.

The stock price of QURE has dropped by 56.98%, falling from $67.69 to $29.12 as of 11:21 AM EST.

uniQure N.V., QURE

The sharp decline in stock price follows unexpected feedback from the U.S. Food and Drug Administration (FDA) regarding the company’s gene therapy AMT-130. This therapy, which is under investigation for treating Huntington’s disease, has faced a significant hurdle in its path to approval, causing a dramatic shift in investor sentiment.

FDA’s Unexpected Feedback Delays BLA Submission for AMT-130

QURE received surprising feedback from the FDA during a pre-Biologics License Application (BLA) meeting concerning AMT-130. The FDA communicated that the data from the ongoing Phase I/II studies, which compared results to an external control, may no longer be sufficient for BLA submission. The company had previously discussed this approach with the FDA in prior meetings, and the data had been expected to serve as primary evidence for the BLA filing under the Accelerated Approval pathway.

This unexpected shift in FDA’s stance has caused significant uncertainty regarding the timing of AMT-130’s potential approval. UniQure had anticipated a smoother path to filing, based on prior communications with the agency. The company now faces the challenge of determining how to proceed with its application and will need to work closely with the FDA to explore alternative strategies for submitting AMT-130 for approval.

AMT-130 Faces Setback Despite Breakthrough Therapy Designation

AMT-130 had previously been granted Breakthrough Therapy designation by the FDA in April 2025, based on its potential to address unmet medical needs in Huntington’s disease. This designation had raised hopes for accelerated approval, especially as the therapy targets a condition with no disease-modifying treatments available. AMT-130 received Regenerative Medicines Advanced Therapy (RMAT) designation from the FDA in May 2024, which further underscored the promise of the therapy.

Despite these designations, the recent feedback from the FDA has put a significant damper on the timeline for AMT-130’s approval. UniQure remains committed to working with the agency to find a viable path forward. The company plans to continue discussions with other regulatory bodies, including those in the European Union and the United Kingdom, in hopes of advancing AMT-130’s approval process outside the U.S.

UniQure’s Commitment to Huntington’s Disease Treatment Remains Strong

QURE’s commitment to advancing AMT-130 as a treatment for Huntington’s disease remains unwavering. The company continues to believe in the potential of AMT-130 to offer significant benefits to patients suffering from this debilitating condition. The recent feedback from the FDA has been a surprise, and the company expressed its disappointment over the news, particularly given the lack of disease-modifying treatment options available for Huntington’s disease patients.

QURE has expressed its intention to urgently engage with the FDA to address the concerns raised and determine the best path forward. The company remains focused on delivering AMT-130 to patients in need and is determined to overcome the regulatory hurdles. In the meantime, uniQure will continue to work with other regulatory agencies to explore potential approval pathways for the gene therapy in different markets.

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