The US Food and Drug Administration (FDA) has ruled that data from Phase I/II studies are insufficient evidence to approve the marketing authorisation for uniQure’s AMT-130, an investigational gene therapy for Huntington’s disease (HD).  

In response, investors have issued a class action securities lawsuit to recover losses. 

The FDA said that the Phase I/II studies were not sufficient to provide the primary evidence of effectiveness and strongly recommended uniQure conduct a prospective, randomised, double-blind, sham surgery-controlled study. However, many in the industry have questioned the ethics of ‘sham’ surgery.  

uniQure has stated that it intends to continue engaging with the FDA regarding Phase III development considerations and plans to request a Type B meeting in the second quarter of 2026 to further discuss potential study design approaches. 

“While we did not reach alignment on a submission pathway based on the Phase I/II data, we believe the totality and durability of our data warrant continued substantive dialogue regarding how the FDA’s stated commitment to regulatory flexibility may be appropriately applied in this setting,” said Matt Kapusta, Chief Executive Officer at uniQure. “We remain committed to engaging with the FDA to determine a clear, scientifically grounded, and efficient path forward for AMT-130.” 

Following the announcement, a class action securities lawsuit was launched by company investors claiming that uniQure made false statements about its prospects. They claim that the company concealed that the design of the pivotal study—including comparison of the pivotal study results to the ENROLL-HD external historical data set—was not fully approved by the FDA. 

They also claim that uniQure downplayed the likelihood that, despite purportedly highly successful results from the pivotal study, uniQure would have to delay its Biologics License Application timeline to perform additional studies to supplement its BLA submission.