UniQure said the FDA signaled it no longer regards data from the company’s Phase I/II AMT-130 program versus an external control as sufficient to support a Biologics License Application, throwing the previously planned 2026 filing into uncertainty. The agency’s apparent shift contrasts with earlier interactions in which uniQure believed it had alignment on using the external-control analysis. Shares plunged as investors priced in a setback to a high-profile gene therapy program. UniQure said it will seek minutes from the pre-BLA meeting and urgently engage the agency to identify a path forward. The company described the decision as an unexpected reversal of prior guidance. Regulatory watchers view the FDA’s stance as emblematic of rising skepticism about single-arm or external-control evidence for complex gene therapies. Biotechs relying on similar evidence frameworks should anticipate tougher discussions with regulators and potential demands for randomized or additional controlled data.