United States Food and Drug Administration Rebukes uniQure

UniQure N.V. is facing a significant crisis due to public rebukes from the United States — Food and Drug Administration and the United States — United States Department of Health and Human Services regarding its lead gene therapy candidate, AMT-130, for Huntington s disease. United States — Food and Drug Administration officials have called AMT-130 a 'failed therapy' and accused UniQure of presenting 'distorted or manipulated comparison' data and mischaracterizing the agency's requests for clinical study design, particularly concerning sham surgeries. The United States — Food and Drug Administration also disputed the eligibility of AMT-130 for streamlined rare-disease pathways and stated it never approved the use of the ENROLL-HD external historical data set as a primary control. These revelations, which began on November 3, 2025, led to a 49% drop in UniQure's stock price and have since caused a nearly 84% decline. A securities class action lawsuit, Ignacio Scocco v. UniQure N.V., et al., has been filed by Hagens Berman, alleging that UniQure misrepresented its interactions with the United States — Food and Drug Administration and downplayed the necessity of a sham-controlled surgery arm for Phase III trials, misleading investors about the Biologics license application timeline.