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Durable Bleed Protection for All Bleeding Subtypes and Favorable Safety Over 5 Years (end-of-study) in the Phase 3 HOPE-B Trial for Persons With Severe or Moderately Severe Haemophilia B

Durable Bleed Protection for All Bleeding Subtypes and Favorable Safety Over 5 Years (end-of-study) in the Phase 3 HOPE-B Trial for Persons With Severe or Moderately Severe Haemophilia B
Highlights From the 19th Annual Congress of the European Association for Haemophilia and Allied Disorders 2026

Durable Bleed Protection for All Bleeding Subtypes and Favorable Safety Over 5 Years (end-of-study) in the Phase 3 HOPE-B Trial for Persons With Severe or Moderately Severe Haemophilia B

Presenting Author: Wolfgang Miesbach, MD, PhD

REVIEW ABSTRACT

F. W. G. Leebeek,1 W. Miesbach,2 M. Recht,3,4 N. S. Key,5 G. Castaman,6 S. Lattimore,7 M. Coppens,8,9 N. O’Connell,10 S. Le Quellec,11 N. W. D. Jansen,11 F. Wang,11 D. Drelich,11 P. E. Monahan,11 S. W. Pipe12 on behalf of all HOPE-B investigators

  1. Erasmus University Medical Center Rotterdam, Rotterdam, Netherlands

  2. University Hospital Frankfurt, Frankfurt, Germany

  3. Yale School of Medicine, New Haven, CT

  4. National Bleeding Disorders Foundation, New York, NY

  5. University of North Carolina, Chapel Hill, NC, United States

  6. Center for Bleeding Disorders and Coagulation, Department of Heart, Lungs, and Vessels, Careggi University Hospital, Florence, Italy

  7. Oregon Heath & Science University, Portland, OR, United States

  8. Department of Vascular Medicine, Amsterdam UMC, University of Amsterdam

  9. Amsterdam Cardiovascular Sciences, Pulmonary Hypertension & Thrombosis, Amsterdam, Netherlands

  10. National Coagulation Centre, St. James’s Hospital; School of Medicine, Trinity College, Dublin, Ireland

  11. CSL Behring, King of Prussia, PA

  12. Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, MI, United States

At the EAHAD Congress, 5-year data from the Phase 3 HOPE-B trial of etranacogene dezaparvovec were presented. Among 54 adult participants with severe or moderately severe haemophilia B, etranacogene dezaparvovec achieved durable bleed protection across all bleeding subtypes, with the annualized bleeding rate (ABR) decreasing from 3.61 pretreatment to 1.25 post-infusion. Factor IX activity remained stable, averaging 36.1 IU/dL at year 5, supporting a 96% reduction in exogenous factor IX use. Most participants (94%) remained free from prophylaxis over 5 years. Etranacogene dezaparvovec demonstrated a favorable safety profile, with early, transient ALT elevations as the most common treatment-related adverse events and no factor IX inhibitors, thrombotic events, late hepatotoxicity, or vector-related oncogenicity. Long-term monitoring will continue for up to 15 years to assess durability and safety.

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

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