Uniqure NV (NASDAQ: QURE) shares plummeted more than 58% in early trading on Monday after the gene therapy company revealed that it was told by the US Food and Drug Administration (FDA) the clinical data for its gene therapy for Huntington's disease is inadequate.
Uniqure said the FDA no longer agrees that data from Phase I/II trials, when compared with external control datasets, would be sufficient to support a Biologics License Application (BLA).
The company's stock price shot up nearly 200% in September after Uniqure reported trial data that showed its gene therapy, AMT-130, slowed progression of the brain disorder by 75%.
“We are surprised by the FDA’s feedback at the recent pre-BLA meeting, which is a drastic change from the guidance the FDA provided in November 2024 that data from the ongoing Phase I/II studies, compared to a natural history external control, may serve as the primary basis for a BLA submission under the Accelerated Approval pathway,” Uniqure CEO Matt Kapusta said in a statement.
“We strongly believe that AMT-130 has the potential to bring substantial benefit to patients, and we remain fully committed to working with the FDA to determine the best path forward to rapidly bring AMT-130 to patients and their families in the US.”
Uniqure noted that it plans to urgently interact with the FDA to find a path forward for the timely accelerated approval of AMT-130.