Efficacy and Safety of Fidanacogene Elaparvovec in Adults With Moderately Severe or Severe Hemophilia B: Results From the Phase 3 BENEGENE-2 Gene Therapy Trial

Efficacy and Safety of Fidanacogene Elaparvovec in Adults With Moderately Severe or Severe Hemophilia B: Results From the Phase 3 BENEGENE-2 Gene Therapy Trial
Highlights From the 31st Congress of ISTH

Presented by: Adam Cuker, MD, MS, Department of Medicine and Department of Pathology & Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA

Efficacy and Safety of Fidanacogene Elaparvovec in Adults With Moderately Severe or Severe Hemophilia B: Results From the Phase 3 BENEGENE-2 Gene Therapy Trial

Presenting Author: Adam Cuker, MD, MS, Department of Medicine and Department of Pathology & Laboratory Medicine, Perelman School of Medicine, University of Pennsylvania, Philadelphia, Pennsylvania, USA

Co-Authors:

Hazzaa Alzahrani – Adult Haematology, King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia

Jan Astermark – Department of Hematology Oncology and Radiation Physics, Skåne University Hospital, and Institution of Translational Medicine, Lund University, Malmö, Sweden

Laurent Frenzel – Department of Haematology, Institut Necker, Paris, France

Olga Katsarou – Blood Transfusion Centre, National Reference Centre for Congenital Bleeding Disorders, Laiko General Hospital, Athens, Greece

Kaan Kavakli – Department of Hematology, Ege University Children’s Hospital, Izmir, Turkey

Robert Klamroth, MD, PhD (he/him/his) – Vivantes Klinikum im Friedrichshain, Clinic for Internal Medicine Angiology and Haemostaseology, Berlin, Germany

Davide Matino, MD (he/him/his) – Department of Medicine, Division of Hematology, McMaster University, Hamilton, ON, Canada 

Amy Shapiro – Indiana Hemophilia & Thrombosis Center, Indianapolis, IN, USA

Jiaan-Der Wang – Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan

Joanne Fuiman – Pfizer Inc.

John McKay – Pfizer Inc.

Pengling Sun – Pfizer Inc. 

Jeremy Rupon, MD,PhD – Pfizer Inc.

 

Reduction in ABR Post-infusion

Reduction in ABR Post-infusion

The percentage reduction and P value were obtained from a repeated measures generalized linear model with negative binomial distribution and log link function. ABRtotal=annualized bleeding rate for total treated and untreated bleeds; FIX=factor IX

Stable FIX Activity up to 24 Months Post-infusion

Stable FIX Activity up to 24 Months Post-infusion

Boxplots show minimum, Q1, median, Q3, and maximum; plus signs indicate mean. Mean (SD) FIX activity values shown above bars. Dotted lines represent mild disease severity range (5–40%) as per the World Federation of Hemophilia definition. FIX=factor IX; max=maximum; min=minimum; Q=quartile

Results from the single-arm, phase 3 pivotal BENEGENE-2 trial for up to 15 months post-infusion of fidanacogene eleparvovec in male patients with severe or moderately severe hemophilia B. Gene infusion resulted in a 71% decrease in mean ABR from week 12 to month 15 (right panel). In addition, 64% of participants had no bleeding events during this post-infusion period. Mean FIX activity (based on a one-stage assay) was stable over a 24-month post-infusion period (left panel), ranging from 28% (week 12) to 25% (month 24).

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Supported through educational grants from BioMarin, CSL Behring, Pfizer Inc., and Spark Therapeutics

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