The following is an excerpt from a press release from uniQure. Read the update in its entirety.
Today uniQure announced a clinical hold by the U.S. Food and Drug Administration (FDA) in its phase III HOPE-B: Trial of AMT-061 in Severe or Moderately Severe Hemophilia B Patients.
Per uniQure’s statement:
“The clinical hold was initiated following the submission of a safety report in mid-December relating to a possibly related serious adverse event associated with a preliminary diagnosis of hepatocellular carcinoma (HCC), a form of liver cancer, in one patient in the HOPE-B trial that was treated with etranacogene dezaparvovec (AMT-061) in October 2019. The patient has multiple risk factors associated with HCC, including a twenty-five-year history of hepatitis C (HCV), hepatitis B virus (HBV), evidence of non-alcoholic fatty liver disease and advanced age. Chronic infections with hepatitis B and C have been associated with approximately 80% of HCC cases.1
The liver lesion was detected during a routine abdominal ultrasound conducted as part of the required study assessments in patients at one-year post dosing. A full surgical resection of the lesion is scheduled this week that will allow for confirmation of the diagnosis. No other events of HCC have been noted in uniQure clinical trials conducted in nearly 80 patients in hemophilia B and other indications, with long-term observation of up to five years in a handful of patients.”
Additionally, uniQure management along with Dr. Steven Pipe, principal investigator in the pivotal, Phase III HOPE-B trial, and Dr. Graham Foster, professor of hepatology at Queen Mary University of London, UK, will host a conference call today, Monday, December 21, 2020, at 8:30am ET. The conference call may be accessed by dialing (877) 870.9135 for domestic callers and +44 020 719 283 38 for international callers. The passcode is 5892217. Please specify to the operator that you would like to join the “uniQure Conference call.” The webcast may also be accessed through the Investors section of the uniQure website. Following the live webcast, a replay of the call will be archived for several weeks.
Patients who are enrolled in the clinical trial will continue to be under close clinical observation according to study protocol.
Patient dosing is complete in each of uniQure’s three hemophilia B gene therapy studies, and there is no plan to enroll or treat additional patients.
As has been our past practice (and consistent with the principles articulated at the January 2020 Safety Summit), NHF and HFA commit to keeping the bleeding disorders community informed as we gather further information. Community members are encouraged to submit your questions and concerns to our joint Safety Inbox.