UniQure said a recent FDA meeting signaled the agency no longer supports relying on its Phase I/II AMT‑130 data against an external control as sufficient for a BLA submission, casting the timing of any approval filing into doubt. The agency’s shift reverses prior discussions in which uniQure believed its external‑control strategy and three‑year data would enable a submission. The company’s shares plunged more than 50% on the news as investors digested the regulatory setback and the potential need for additional data generation. The episode amplifies uncertainty about the FDA’s current stance on external controls and real‑world evidence for high‑profile gene therapies, a development that will influence other developers planning accelerated pathways based on single‑arm or externally controlled studies.