Highlights From the 17th Annual Congress of EAHAD
Seven-year follow-up of valoctocogene roxaparvovec gene therapy for haemophilia A
Presenting Author: Glenn F. Pierce, MD, PhD
E. Symington1, S. Rangarajan2, W. Lester3, B. Madan4, G. F. Pierce5, P. Raheja6, C. Millar7, T. M. Robinson8
- Cambridge University Hospitals NHS Foundation Trust, Cambridge
- Faculty of Medicine, University of Southampton, Southampton
- University Hospitals Birmingham NHS Foundation Trust, Birmingham
- Guy’s and St Thomas’ NHS Foundation Trust, London, United Kingdom
- Independent Consultant, La Jolla, United States
- Haemophilia Centre Royal London Hospital, Barts Health NHS Trust
- Imperial College Healthcare NHS Trust and Centre for Haematology, Department of Immunology and Inflammation, Imperial College London, London, United Kingdom
- BioMarin Pharmaceutical Inc., Novato, United States
Data Points
Figure 1: Schematic of Clinical Trial
Adult males ≥ 18 years with severe hemophilia A, who were previously receiving exogenous FVIII and had no history of FVIII inhibitors or anti-AAV5 antibodies, received an infusion of 6 x 1013 (n = 7) or 4 x 1013 (n = 6) vg/kg valoctocogene roxaparvovec.
Figure 2: Sustained Reduction in Annualized Bleeding Rate (ABR)
This figure shows the 7-year ABRs in patients who received either 6x1013 vg/kg or 4x1013 vg/kg of valoctogene roxaparvovec. At each yearly follow-up, mean ABRs decreased from baseline by 96% for the 6x1013 and 88% for the 4x1013 cohorts at years 7 and 6, respectively.
Figure 3: Sustained Reduction in Annualized FVIII Infusion Rate
This figure shows the 7-year annualized FVIII infusion rate in patients who received either 6x1013 vg/kg or 4x1013 vg/kg of valoctogene roxaparvovec. The mean annualized FVIII infusion rate for the 6 x 1013 and 4 x 1013 cohorts were 6.4 (1.6) and 9.3 (5.1) infusions/year over all follow-up, representing a decline of 95% and 93% from baseline, respectively.
Figure 4: FVIII Activity Over 7 Years
This figure shows the 7-year annualized FVIII activity in patients who received either 6x1013 vg/kg or 4x1013 vg/kg of valoctogene roxaparvovec. FVIII activity has declined in both cohorts over 7 years, however, the rate of decrease is lower in each following timepoint.
Figure 5: Individualized Participant FVIII Activity per Chromagenic Substrate Assay (CSA)
This figure shows the individual 7-year annualized FVIII activity in patients who received either 6x1013 vg/kg or 4x1013 vg/kg of valoctogene roxaparvovec. There is a decrease in FVIII activity in both cohorts over 7 years.
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