uniQure announced on June 17 that it plans to submit a biologics license application for its Huntington’s disease gene therapy in the third quarter, following the Food and Drug Administration's agreement that three-year data from its Phase 1/2 trial can support accelerated approval.
Stifel wrote in a note to investors, “This is obviously a ~180 from FDA.” Shares of uniQure rose 62% at market open on Wednesday to $44 after the update from its Type B meeting with the FDA. The company’s stock had begun rising early last month amid rumors of former FDA commissioner Marty Makary's departure, who had been critical of the therapy.
The agency will not necessarily require a sham surgery design for what uniQure called a confirmatory study of AMT-130. Previously, the FDA insisted on an additional sham surgery–controlled Phase 3 trial before considering AMT-130 for approval—a design some experts and advocacy groups argue is unethical. Now, according to uniQure’s announcement, the agency is considering using standard-of-care control instead. The company said, “the FDA seeks to align on the confirmatory study design prior to the BLA submission,” and added that “FDA communicated that they would work as expeditiously as possible with uniQure on this effort.”
If approved, AMT-130 would be the first genetic treatment for Huntington’s disease. In a prepared statement Wednesday, uniQure CEO Matt Kapusta said, “Today’s announcement reflects the outcome we have worked toward throughout our continued regulatory engagement with FDA, and we are deeply grateful for FDA’s genuine commitment to addressing the unmet need of Americans living with Huntington’s disease.”
The regulatory process has been challenging for uniQure. The company originally intended to seek approval in early 2026 based on three-year data but was told by regulators during a pre-BLA meeting in November 2025 that these data were no longer considered adequate primary evidence. This came despite previous agreements between both parties regarding protocols and statistical analyses.
Analysts have compared this development with recent instances where other biotechs such as Capricor Therapeutics and Biohaven also saw reversals in previous Food and Drug Administration guidance under recent leadership changes at the agency.
