Wolfgang Miesbach: Excellent 4-Year Data of Etranacogene Dezaparvovec in Haemophilia B
Wolfgang Miesbach, Professor of Medicine at Frankfurt University Hospital, shared a post on LinkedIn:
”Excellent 4-Year Data of Etranacogene Dezaparvovec in Haemophilia B: The Subgroup (N=33) With No Neutralizing Anti-AAV Antibodies (NAb-Negative):
- 100% expressed endogenous FIX at therapeutic levels (mean 39.0 IU/dL)
- 100% remained prophylaxis-free over 4 years—zero returned to prophylaxis
- Exogenous FIX consumption decreased by 99%
- 85% reduction in bleeds (all, spontaneous, joint)
- Safety: Treatment-related AEs only first 3 months; transient ALT elevations 18%, all manageable
This enables direct comparison to other haemophilia B gene therapies where only NAb-negative patients were enrolled.
HOPE-B’s Inclusive Design
HOPE-B is the first—and currently only—haemophilia B gene therapy trial to include patients with pre-existing AAV neutralizing antibodies for treatment with etranacogene dezaparvovec.
Very recently, the NEJM paper presented 5-year data of the full cohort (NAb-negative and NAb-positive; N=54).
Congratulations to Priyanka Raheja and co-authors!”
Read the full article here.
Article: Etranacogene dezaparvovec in participants with hemophilia B and without adeno-associated virus serotype 5 neutralizing antibodies: A 4-year subgroup analysis (HOPE-B)
Authors: Priyanka Raheja, Niamh O’Connell, Peter Verhamme, Peter Kampmann, Richard S. Lemons, Fei Wang, Sean Gill, Paul E. Monahan, Sandra Le Quellec, Frank W. G. Leebeek
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