Amsterdam-based uniQure has announced that its hemophilia B gene therapy program, including its phase 3 HOPE-B study, has been placed on clinical hold by the U.S. FDA.
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.
Etranacogene dezaparvovec consists of an AAV5 viral vector carrying a gene cassette with the patent-protected Padua variant of Factor IX (FIX-Padua). The gene therapy has been in trials for hemophilia B in the hope of finding a possible cure for the bleeding disease.The treatment has been granted Breakthrough Therapy Designation by the US FDA and access to Priority Medicine (PRIME) regulatory initiative by the EMA.
uniQure says the affected patient has multiple risk factors associated with HCC, and no other cases of HCC have been reported in the clinical trials conducted in more than 100 patients in hemophilia B and other indications, with some patients dosed more than 10 years ago.
The drugmaker does not anticipate that the temporary hold will impact the regulatory submission timeline for the hemophilia B program.
Read the press release