On October 29 2025, uniQure held a pre‑Biologics License Application meeting with the U.S. Food and Drug Administration to discuss its Huntington’s disease gene therapy, AMT‑130. The company received the final minutes of that meeting on December 4 2025, which confirmed that the Phase I/II data are unlikely to provide the primary evidence required for a BLA submission.
The FDA’s assessment signals a major regulatory hurdle for uniQure’s lead program. The company had planned to file a BLA in the first quarter of 2026, but the minutes now indicate that the data set is insufficient, potentially delaying the filing and extending the development timeline. UniQure has stated it will urgently request a follow‑up meeting with the FDA in the first quarter of 2026 to define a new path forward.
Financially, uniQure reported a Q3 2025 net loss of $80.5 million, a sharp increase from the $44.4 million loss reported in Q3 2024. Revenue rose to $3.7 million from $2.3 million year‑over‑year, driven primarily by license income. The widened loss was driven by higher non‑operating expenses—including adverse foreign‑currency movements and changes in the fair value of warrant liabilities—as well as increased research and development and selling, general, and administrative costs associated with preparing for a potential commercialization of AMT‑130.
Beyond AMT‑130, uniQure’s pipeline continues to progress. Enrollment in the AMT‑260 epilepsy study is advancing, with data expected in the first half of 2026. Early results from the AMT‑191 Fabry disease study show promising enzyme increases, and data are anticipated in H1 2026. The AMT‑162 ALS study has been paused following a dose‑limiting toxicity event, highlighting ongoing safety challenges in other programs.
CEO Matt Kapusta emphasized the company’s commitment to working with the FDA, noting surprise and disappointment at the feedback and reaffirming confidence in AMT‑130’s potential to transform Huntington’s disease treatment. Chief Medical Officer Walid Abi‑Saab highlighted the positive topline data from early studies, underscoring the therapeutic promise of the platform.
Analysts have shifted focus to regulatory risk following the FDA’s assessment, revising their outlooks on uniQure’s near‑term prospects. The regulatory setback has prompted a reassessment of the company’s development timeline and potential revenue trajectory.
The market reaction has been negative, with investors revising expectations and expressing concern over the regulatory uncertainty surrounding AMT‑130.
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