Steven Pipe, MD, on Continuing Durability of Etranacogene Dezaparvovec in Hemophilia B

"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; 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.

Click here to read more coverage of the ASH 2022 meeting.

REFERENCE

Pipe SW, Leebeek FWG, Recht M, et al. Adults with Severe or Moderately Severe Hemophilia B Receiving EtranacogeneDezaparvovec in the HOPE-B Phase 3 Clinical Trial Continue to Experience a Stable Increase in Mean Factor IX Activity Levels and Durable Hemostatic Protection after 24 Months’ Follow-up. Presented at: 64th ASH Annual Meeting, December 10-12, 2022, in New Orleans, Louisiana. Abstract #2141