The professor of pediatric hematology/oncology at CS Mott Children’s Hospital discussed new follow-up data from the HOPE-B study presented at ASH 2022.
"From month 18 to month 24 of follow-up, we're seeing stable FIX expression and no new adverse events. And so again, this is confirming what we thought going into the trial based on earlier trial experience, and I think it bodes well for the future. Perhaps from this 1-time treatment, we will be able to achieve sustained FIX expression that is potentially transformative for the patient for bleed control and durable for, hopefully, their lifespan.”
Etranacogene dezaparvovec (Hemgenix; EtranaDez; CSL Behring, UniQure) continued to show durable factor 9 (FIX) expression in people with moderate to severe hemophilia B in follow-up data from the phase 3 HOPE-B trial (NCT03569891). Hemgenix was approved for this indication in November 2022.
The data were presented at the 64th American Society of Hematology (ASH) Annual Meeting, held December 10-12, 2022, in New Orleans, Louisiana, by Steven Pipe, MD, professor, pediatric hematology/oncology at CS Mott Children’s Hospital.
CGTLive spoke with Pipe to learn more about the results seen in the HOPE-B trial, which previously met its primary end point of reduction in annualized bleeding rate (ABR) post-treatment compared with baseline FIX prophylactic therapy, as well as new follow-up data that supports the therapy’s durability.