FDA Says AMT-130 Data Inadequate; Shares Tumble

The FDA told UniQure that clinical data from its Phase I/II AMT-130 studies using external controls are inadequate to support a Biologics License Application, reversing earlier guidance. UniQure said the agency conveyed the position after a pre‑BLA meeting and that the change departs from guidance given in November 2024. The agency’s stance leaves the timing of any BLA submission unclear, and UniQure said it will urgently work with the FDA and other regulators to try to establish a path to approval, including potentially accelerated options. The announcement sent U.S.-listed UniQure shares tumbling about 67–68% in premarket trading, erasing much of the stock’s recent gains. Earlier trial data had shown AMT-130 slowed Huntington’s disease progression by roughly 75%, and UniQure’s CEO called the FDA feedback “surprising.”