FDA Lifts Hold on uniQure’s Hemophilia B Gene Therapy Program

The FDA has lifted a clinical hold on uniQure’s hemophilia B gene therapy program, after reviewing a report of a serious adverse event during a phase 3 trial.

The agency initiated the hold in December following a diagnosis of hepatocellular carcinoma (HCC) in a patient with previous risk factors for the disease, including having a history of hepatitis C and hepatitis B infections.

The investigation found that AMT-061 (etranacogene dezaparvovec) was “very unlikely” to have contributed to the patient’s HCC, said Ricardo Dolmetsch, uniQure’s president of research and development. The company plans to release the trial data later this quarter.