Highlights From the ISTH 2024 Congress
Efficacy and Safety of Fidanacogene Elaparvovec in Adults with Moderately Severe or Severe Hemophilia B: Updated Results From the Phase 3 BENEGENE-2 Gene Therapy Trial
Presenting Author: Adam Cuker, MD, MS
Wang JD,1 Cuker A,2 Alzahrani A,3 Astermark J,4 Frenzel L,5 Katsarou O,6 Kavakli K,7 Klamroth R,8 Matino D,9 Shapiro A,10 Kalac M,11 Fuiman J,11 McKay J,11 Sun P,11 Rupon J,11 Plonski F,11
- Center for Rare Disease and Hemophilia, Department of Pediatrics, Taichung Veterans General Hospital Taichung, Taichung, Taiwan (Republic of China)
- Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA
- King Faisal Specialist Hospital and Research Centre Riyadh, , Riyadh, Ar Riyadh, Saudi Arabia
- Department of Translational Medicine, Lund University, Malmö, Skane Lan, Sweden
- Department of Haematology, Institut Necker, Paris, France, Paris, Ile-de-France, France
- Head of the Blood Transfusion Unit, Laiko Hospital, Blood Transfusion Centre, National Reference Centre for Congenital Bleeding Disorders, Athens, Attiki, Greece
- Division of Hematology, Department of Pediatrics, Ege University Faculty of Medicine, Izmir, Turkey
- Head of Department, Vivantes Klinikum, Friedrichshain, Berlin, Germany
- Division of Hematology & Thromboembolism, Department of Medicine, McMaster University, Ontario, Canada
- Indiana Hemophilia & Thrombosis Center, Inc., Indianapolis, IN, USA
- Pfizer Inc., Collegeville, PA, USA
Data Points
Figure 1: Annualized Bleeding Rates (ABR)
This shows annualized bleeding rates (ABR) up to 15 months after infusion in patients who received fidanacogene elaparvovec. Mean ABR decreased from 4.4 during the observation period to 1.3 following gene therapy (71% reduction). This met criteria for both non-inferiority and superiority of fidanacogene elaparvovec compared with factor prophylaxis. 64.4% of subjects had no bleed events from week 12 to month 15 post-infusion.
Figure 2: FIX Activity
The following figure shows FIX activity levels up to 24 months after infusion in patients who received fidanacogene elaparvovec. FIX levels rose to a mean of 27.8% at 12 weeks and remained stable through month 24 as measured by a one-stage SynthASil assay.
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