Clinical Hold Lifted on UniQure Gene Therapy

FDA has removed a clinical hold against UniQure’s hemophilia B gene therapy program after determining it satisfactorily addressed all issues identified by the agency concerning a single patient diagnosed with hepatocellular carcinoma (HCC) in the HOPE-B pivotal trial, according to the company. “Our comprehensive investigation showed that AMT-061 (etranacogene dezaparvovec) is very unlikely to have contributed to the HCC in our patient,” it says.

UniQure says the patient in question had multiple risk factors associated with HCC, including a 25-year history of hepatitis C and a history of hepatitis B. Such chronic infections have been associated with approximately 80% of HCC cases, it says.

Etranacogene dezaparvovec consists of an AAV5 viral vector carrying a gene cassette with the patent-protected Padua variant of Factor IX. The product has been granted a breakthrough therapy designation by FDA. Top-line 52-week data from the trial are expected later this quarter.