Data from uniQure’s Phase I/II AMT-130 not enough, FDA confirms

Gene therapy company uniQure (Nasdaq: QURE) last week received final minutes from the US Food and Drug Administration (FDA) regarding its pre-Biologics License Application (BLA) meeting held in late October to discuss the company’s application for AMT-130 in Huntington’s disease (HD).  

Shares in the Dutch firm closed more than 10% lower on the day as investors digested confirmation of a setback for the development of AMT-130 in the fatal neurodegenerative disease.

In the final meeting minutes, and consistent with uniQure’s press release issued in early November, the FDA conveyed that data submitted from the Phase I/II studies of AMT-130 are currently unlikely to provide the primary evidence to support a BLA submission.