Steven W. Pipe, MD, on Confirming Efficacy, Safety of Hemgenix Gene Therapy in Hemophilia B Populations

“We had concerns about patients who had history of hepatitis. But in this trial, we did have fairly strict criteria for not having any advanced liver fibrosis, and they obviously couldn't have cirrhosis.So,I think that aspect of liver health is still going to be an important screening criterion for evaluating suitability of this product, even in the commercial phase.”

Etranacogene dezaparvovec (CSL Behring/uniQure), approved as Hemgenix for treating severe or moderately severe hemophilia B with or without preexisting AAV5 neutralizing antibodies (NAbs), over standard of care factor IX prophylaxis, has continued to demonstrate efficacy and safety.

Recent 3-year follow-up data of the phase 3, open-label, single-arm HOPE-B trial (NCT03569891) were presented by Steven W. Pipe, MD, a professor of pediatric hematology/oncology at CS Mott Children’s Hospital, at the 2023 American Society of Hematology (ASH) Annual Meeting & Exposition, held December 9-12, in San Diego, California.

CGTLive® spoke with Pipe to learn more about the 3-year follow-up data, as well as asub analysis of the study that focused on patients with hepatitis. He also discussed another research he would like to see done with hemophilia gene therapy, including treating pediatric patients. He also shared his excitement for nonviral and gene editing approaches in the field.

REFERENCE

Pipe S, van der Valk P, Verhamme P, et al. Long-term bleeding protection, sustained FIX Activity, reduction of FIX consumption and safety of hemophilia B gene therapy: Results from the HOPE-B trial 3 years after administration of a single dose of etranacogenedezaparvovecin adult patients with severe or moderately severe hemophilia B. Presented at: ASH 2023 Annual Meeting & Exposition. December 9-12; San Diego, CA. Abstract 1055