uniQure, a prominent gene therapy company, has provided a crucial update concerning its investigational gene therapy AMT-130 for Huntington’s disease (HD). The company received feedback from the U.S. Food and Drug Administration (FDA) during a pre-Biologics License Application (BLA) meeting, indicating a significant shift in the FDA’s stance on the adequacy of data from the Phase I/II studies. Although the final details from the meeting are not yet available, discussions indicated that the FDA may no longer consider the existing data sufficient for supporting a BLA submission.
This change in direction is particularly unexpected, as prior communications during previous meetings with the FDA suggested that the Phase I/II trial data, which included comparisons to a natural history external control, could serve as the primary basis for a BLA submission under the Accelerated Approval pathway. This new feedback leaves the timeline for the BLA submission unclear, creating uncertainty for the potential approval of AMT-130.
In response to the FDA’s comments, uniQure is prepared to engage with the agency to determine the best path forward for accelerating AMT-130’s approval process. CEO Matt Kapusta expressed disappointment over the FDA’s shift but reiterated the company’s commitment to facilitating a treatment option for individuals affected by Huntington’s disease, who currently have no disease-modifying therapies available. “We strongly believe that AMT-130 has the potential to bring substantial benefit to patients,” he affirmed, highlighting the urgency of their mission.
Additionally, while maintaining efforts with the FDA, uniQure intends to pursue dialogue with regulatory agencies in the European Union and the United Kingdom, positioning its investigational treatment on broader international fronts.
AMT-130 has previously been granted breakthrough therapy designation by the FDA, reflecting the agency’s acknowledgment of its potential advantages over existing therapies. Such designations are critical for expediting the review process for promising therapies intended for serious conditions.
uniQure’s ongoing advancements in gene therapy represent a significant step forward for patients with severe diseases, and its persistence in navigating regulatory channels highlights the company’s dedication to improving patient outcomes.
With ongoing engagement with regulatory bodies and a scientific approach underscored by clinical data, uniQure aims to ensure that AMT-130—a potential game-changer for Huntington’s disease—can swiftly reach those in need. As the situation evolves, the commitment to delivering innovative therapies continues to shine through, offering hope to patients and families affected by this challenging condition.