UniQure Asserts Hemophilia B Gene Therapy Is Not Cause of Liver Cancer Case
An independent investigation has shown that uniQure’s hemophilia B gene therapy (etranacogene dezaparvovec) was not responsible for a reported case of liver cancer that led to a clinical hold by the FDA on its entire clinical development program for the gene therapy, the company said.
The FDA imposed the clinical hold in December after one patient in the company’s pivotal phase 3 HOPE-B trial was diagnosed with liver cancer, and a safety report indicated the diagnosis could be associated with the treatment.
At the time, uniQure noted that the patient diagnosed with liver cancer had a long history of hepatitis C, hepatitis B and nonalcoholic fatty liver disease, which are all contributing risk factors for hepatocellular carcinoma. The analysis by the independent laboratory confirmed that the patient had several genetic mutations that are characteristic of the disease.
The company said it has already shared the new findings with the FDA and plans to hold discussions with the agency aimed at lifting the clinical hold.