uniQure told investors that the FDA indicated data from its Phase I/II AMT‑130 program—benchmarked against an external historical control—may no longer be sufficient to support a Biologics License Application. The company said the agency’s stance represents a reversal from prior Type B meeting guidance and left the timing of a BLA submission unclear. uniQure disclosed the shift following a pre‑BLA meeting and said it is awaiting final meeting minutes within 30 days and will urgently seek further interactions with FDA. The announcement triggered a dramatic selloff in the company’s stock, reflecting investor concern over regulatory risk for one‑time CNS gene therapies. Analysts compared the episode to recent FDA pushback on single‑arm or heterogeneous trial datasets in other filings. uniQure emphasized continued commitment to patients and signaled it will work with regulators to identify a path forward; operational and funding timelines will now hinge on those next steps.