UniQure disclosed that FDA officials indicated the company’s Phase I/II data for AMT‑130 may no longer be adequate to support a Biologics License Application (BLA), reversing earlier agency communications. UniQure said the change follows a pre‑BLA meeting and has put the timing of a U.S. submission into doubt. The company’s September mid‑stage readout had shown large, durable reductions in disease progression and had driven a sharp rally in the stock. FDA’s shift to requiring more evidence forces UniQure to reassess filing strategy and timelines and raises questions about reliance on external‑control comparisons for accelerated pathways. Investors reacted with heavy selloffs, and the company said it will urgently engage the agency to identify a path forward. The development underscores regulatory scrutiny of single‑arm and external control datasets in gene‑therapy submissions.