FDA lifts hold on uniQure hemophilia B gene therapy program

The US Food and Drug Administration (FDA) has removed the clinical hold on uniQure’s (Nasdaq: QURE) hemophilia B gene therapy program after determining that Dutch gene therapy company had satisfactorily addressed all issues identified by the FDA related to a single patient diagnosed with hepatocellular carcinoma (HCC) in the HOPE-B pivotal trial of AMT-061.

The news pushed uniQure’s New York traded shares up 8.4% to $35.00 in pre-market activity today.

“Patient safety is our top priority, and we are grateful to our advisors and the FDA for their help in resolving this clinical hold,” stated Richard Dolmetsch, president of research and development at uniQure. “Our comprehensive investigation showed that AMT-061 (etranacogene dezaparvovec) is very unlikely to have contributed to the HCC in our patient. We look forward to announcing top-line 52-week data from the HOPE-B pivotal trial later this quarter,” he added.