Results from a Phase III trial have confirmed the durability and safety of a one-time infusion to treat adults living with moderate to severe haemophilia B. 

Etranacogene dezaparvovec, developed by global biopharma company CSL, was studied in a Phase III open-label, single-dose, single-arm trial, with 54 male participants infused with a single dose. Of the 54 participants, 50 completed five years of follow-up. 

The findings of the study, published in the New England Journal of Medicine (NEJM), showed etranacogene dezaparvovec reduced annualised bleeding rate by approximately 90%  and 94% of patients remained free of continuous prophylaxis treatment. The results were also confirmed during the American Society of Hematology (ASH) Annual Meeting. 

“The five-year HOPE-B results mark a pivotal milestone for gene therapy, providing clear, five-year data of the ability of etranacogene dezaparvovec to potentially reshape care for adults with haemophilia B,” said Steven Pipe, Professor of Paediatrics and Pathology, Haemophilia and Coagulation Disorders Programme and the Special Coagulation Laboratory, University of Michigan.  

“For those who have relied on frequent prophylactic infusions, achieving years of bleed control from a single treatment offers the potential for greater day-to-day freedom and a life less burdened by the demands of ongoing therapy.” 

More than 75 individuals across eight countries have already received etranacogene dezaparvovec in real-world settings. The treatment has received regulatory approval in the United States, Canada, the UK, Switzerland, Australia, Saudi Arabia, Taiwan, South Korea, Singapore, and Hong Kong, with conditional marketing authorisation from the European Commission (EC) for the European Union and European Economic Area. 

Bruno Quinney, Content Team, DDW