Apr 27, 2021

uniQure recently announced that the U.S. Food and Drug Administration (FDA) has removed a clinical hold on their hemophilia B gene therapy program. The FDA had determined that uniQure satisfactorily addressed all issues raised by the agency that were related to a single patient diagnosed with hepatocellular carcinoma (HCC), a common form of liver cancer.

The patient had been participating in the phase III HOPE-B pivotal trial of etranacogene dezaparvovec, uniQure’s investigational gene therapy candidate for individuals with severe and moderately severe hemophilia B, when a preliminary diagnosis of HCC was reported in December 2020. According to a company press release, the patient had “multiple risk factors” associated with HCC, including a twenty-five-year history of hepatitis C and hepatitis B. Further, chronic infections with hepatitis B and C have been associated with approximately 80% of HCC cases.

The liver is the organ targeted for delivery of etranacogene dezaparvovec which consists of adeno-associated virus serotype 5 (AAV5), which has been demonstrated to be safe and well-tolerated, acting as a delivery vector carrying a gene cassette with the Padua variant of factor IX (FIX). AAV5 is a variant of the type of the adeno-associated virus (AAVs) vectors that are being investigated in multiple gene therapy trials.

uniQure subsequently launched a case investigation, the results of which were announced in a company released last month. Read NHF’s March 30th news release to learn more about the findings of the HCC case investigation.

 “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 Ricardo Dolmetsch, PhD, 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.”

Source: uniQure press release dated April 26, 2021