FDA lifts clinical hold on uniQure gene therapy

April 26, 2021

uniQure announced that the U.S. FDA has removed the clinical hold that was placed on the company's hemophilia B gene therapy program after a patient was diagnosed with a rare form of liver cancer in a late-stage trial.

On December 21, 2020, the FDA placed a hold on the Amsterdam-based drugmaker's hemophilia B program, including its phase 3 HOPE-B pivotal study of etranacogene dezaparvovec for the treatment of patients with severe and moderately severe hemophilia B, after a patient in the trial was diagnosed with hepatocellular carcinoma (HCC).

According to uniQure, a comprehensive investigation showed that the treatment, AMT-061, "is very unlikely to have contributed to the hepatocellular carcinoma in our patient." And the FDA agrees, lifting the hold after concluding that the company satisfactorily addressed all issues identified by the agency.

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.

According to uniQure, who is currently using its validated technology platform to advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases, 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. As a standard precaution, all patients in uniQure’s hemophilia B gene therapy program, including the 54 patients in HOPE-B, have had abdominal ultrasounds performed one year after dosing, and each will continue to be monitored by their care teams. Patients will continue to receive abdominal ultrasounds every six-months.

Read the press release