UniQure Inc has announced positive results from the HOPE-B clinical trial on the efficacy and safety of etranacogene dezaparvovec-drlb (Hemgenix®), the first gene therapy for adults with hemophilia B. The results, announced via news release, have been published in the New England Journal of Medicine.

“HOPE-B was . . . the first and only phase 3 study to demonstrate efficacy of a gene therapy for hemophilia B in individuals with circulating neutralizing antibodies that have the potential to interfere with the effects of treatment,” Steven Pipe, lead investigator of the study and a professor of pediatrics and pathology at the University of Michigan said.

“Results from HOPE-B suggest that Hemgenix may be effective in a broad range of hemophilia B patients, regardless of prior exposure to common adeno-associated viruses.”

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Etranacogene dezaparvovec is a novel gene therapy administered via a single intravenous infusion. The US Food and Drug Administration (FDA) approved it in November 2022 and the European Medicines Agency (EMA) approved it in February 2023.

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The phase 3 HOPE-B trial is a multinational, open-label study that is ongoing. It enrolled 54 adult patients with moderately severe to severe hemophilia B who required prophylaxis with factor IX replacement for 6 months or longer to establish baseline annualized bleeding rates (ABRs). After this, they received a single administration of etranacogene dezaparvovec and were followed for at least 18 months.

The results showed etranacogene dezaparvovec to be superior to routine factor IX prophylaxis in reducing ABR, joint bleeds, and factor IX activity, consumption, and infusion rate. No serious adverse events related to the treatment were reported.

Long-term, 24-month data supporting the durable efficacy and safety of etranacogene dezaparvovec were presented at the 54th American Society of Hematology (ASH) 2022 Annual Meeting and Exposition and the European Association for Haemophilia and Allied Disorders (EAHAD) 2023 Annual Meeting.


UniQure announces HOPE-B clinical trial data published in the New England Journal of Medicine, demonstrating durability and other benefits of Hemgenix® (etranacogene dezaparvovec-drlb). News release. UniQure Inc; February 23, 2023.