UniQure disclosed that the FDA now signals it may no longer accept AMT‑130’s Phase I/II data — based on comparisons to an external control — as sufficient evidence to support a Biologics License Application for its Huntington’s disease gene therapy. The agency’s apparent reversal contrasts with prior communications that had encouraged UniQure’s planned accelerated approval pathway, and the company said the timing of any U.S. submission is now unclear. The shift sent UniQure shares sharply lower and underscores regulatory risk for single‑arm or external‑control strategies in rare‑disease approvals. Sponsors relying on nonrandomized comparators must now reassess evidence packages and potential additional data collection. The case may influence agency‑industry discussions about acceptable endpoints and statistical analysis plans for gene therapies targeting neurodegenerative diseases.