uniQure N.V. has scheduled a Type A meeting with the U.S. Food and Drug Administration to review the Biologics License Application (BLA) data package for its Huntington’s disease gene‑therapy candidate, AMT‑130. The meeting is a formal, pre‑submission discussion that will allow the company to address the FDA’s recent concerns about the sufficiency of its Phase I/II data for accelerated approval, following a reversal on the use of external control data.
The FDA’s concerns stem from a prior pre‑BLA meeting in October 2025, where the agency expressed doubts that the Phase I/II data alone would support accelerated approval. Minutes from that meeting, released in December 2025, highlighted the need for additional evidence or a different analytical approach. The Type A meeting is the next step in a regulatory dialogue that began with the Breakthrough Therapy designation in April 2025 and the Regenerative Medicine Advanced Therapy (RMAT) designation in May 2024.
AMT‑130 has shown a statistically significant, dose‑dependent slowing of disease progression in the interim Phase I/II study, but the FDA now requires more robust data to justify accelerated approval. The company’s strategy is to present the full BLA data package and to discuss potential supplemental studies or alternative control designs that could satisfy the agency’s requirements.
Financially, uniQure reported cash, cash equivalents and investments of $694.2 million as of September 30 2025, giving the company a runway that extends to 2029. This strong liquidity position allows uniQure to fund additional clinical work or regulatory submissions without immediate financing pressure, which is critical given the uncertainty surrounding the approval pathway.
Investors have reacted positively to the announcement, with pre‑market activity indicating optimism about the meeting’s potential to clarify the regulatory path. While the meeting does not guarantee approval, it signals that the FDA is willing to engage in a detailed discussion of the BLA data, which could accelerate the company’s commercial plans if the agency’s concerns are adequately addressed.
CEO Matthew Kapusta said the company is “looking forward to a constructive discussion” and will provide a regulatory update once the official meeting minutes are released, underscoring uniQure’s confidence in its data and its commitment to working closely with the FDA.
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