The FDA told uniQure it does not view the existing single‑arm data package for AMT‑130, a gene therapy candidate for Huntington’s disease, as sufficient for approval and strongly recommended a prospective, randomized, double‑blind, sham‑surgery‑controlled study. UniQure confirmed the agency’s preference after a regulatory meeting and said plans to seek approval remain blocked. The guidance sent uniQure shares sharply lower as investors digested the need for a new randomized trial—an outcome that would substantially increase costs and extend timelines. AMT‑130 is an AAV‑based gene therapy intended to lower mutant huntingtin expression; regulators’ call for a randomized sham‑controlled design underscores FDA expectations for rigorous control arms in single‑procedure CNS gene‑therapy trials.