UniQure Expects To Resolve FDA Clinical Hold Within Three Months

21 Dec 2020

The gene therapy EtranaDez for hemophilia B placed on clinical hold after a patient was diagnosed with liver cancer, but UniQure points to that patient’s extensive history of liver disease.

UniQure's gene therapy for hemophilia B faces a stumbling block

A pivotal Phase III trial for uniQure N.V.’s gene therapy candidate for hemophilia B – etranacogene dezaparvovec (EtranaDez) – has been placed on a clinical hold by the US Food and Drug Administration due to one patient being diagnosed with hepatocellular carcinoma (HCC). However, the company and several analysts think an analysis of the patient’s cancer tissue may show that underlying risk factors for HCC more likely led to the cancer than to the therapy or its adeno-associated virus (AAV) construct.

The Amsterdam-headquartered firm reported the clinical hold before US stock trading opened on 21 December. The patient, a 75-year-old who...