Margareth C. Ozelo, MD, PhD
Highlights From the ISTH 2021 Congress

Efficacy and Safety of Valoctocogene Roxaparvovec Adeno-associated Virus Gene Transfer for Severe Hemophilia A: Results From the Phase 3 GENEr8-1 Trial

M.C Ozelo1, J. Mahlangu2, KJ. Pasi3, A. Giermasz4, A.D Leavitt5, M. Laffan6, E. Symington7, D.V Quon8, J.-D. Wang9, K. Peerlinck11, S. Pipe11, B. Madan12, N.S Key13, G.F Pierce14, B. O’Mahony15,16, R. Kaczmarek17,18, A. Lawal19, M. Huang19, W.Y. Wong19, B. Kim19, GENEr8-1 Trial Group

1Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, Brazil

2Hemophilia Comprehensive Care Center, Charlotte Maxeke Johannesburg Academic Hospital, University of the Witwatersrand and NHLS, Johannesburg, South Africa

3Barts and the London School of Medicine and Dentistry, London, United Kingdom

4Hemophilia Treatment Center, University of California Davis, Sacramento, United States

5University of California, San Francisco, United States

6Centre for Haematology, Imperial College London, London, United Kingdom

7Cambridge University Hospitals NHS Foundation Trust, Cambridge, United Kingdom

8Orthopaedic Hemophilia Treatment Center, Los Angeles, United States

9Center for Rare Disease and Hemophilia, Taichung Veterans General Hospital, Taichung, Taiwan, Province of China

10Department of Vascular Medicine and Haemostasis and Haemophilia Centre, University Hospitals Leuven, Leuven, Belgium

11Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, United States

12Guy's & St. Thomas’ NHS Foundation Trust, London, United Kingdom

13UNC Blood Research Center, University of North Carolina, Chapel Hill, United States

14Independent Consultant, La Jolla, United States

15Irish Haemophilia Society, Dublin, Ireland

16Trinity College, Dublin, Ireland

17Department of Pediatrics, Indiana University School of Medicine, IUPUI-Wells Center for Pediatric Research, Indianapolis, United States

18Laboratory of Glycobiology, Hirszfeld Institute of Immunology and Experimental Therapy, Wroclaw, Poland

19BioMarin Pharmaceutical Inc., Novato, United States

Key Data Points

FVIII Activity Over 52 Weeks in mITT Population

FVIII activity using the chromogenic assay for patients (N = 132) in the phase 3 GENEr8-1 Trial treated with a single infusion of valoctocogene roxaparvovec (6 x 1013 vg/kg). At weeks 49–52, mean (95% CI) change from baseline in FVIII activity was 41.9 (34.1–49.7) IU/dL (P < 0.001), and median (Q1, Q3) change from baseline was 22.9 (10.9, 61.3) IU/dL.

ABR and FVIII Infusions/Year
Annualized bleed rate (ABR, left panel) and number of FVIII infusions/year (right panel) for the rollover population (n = 112) in the GENEr8-1 trial. For ABR, the mean (95% CI) change was a decrease of 4.1 (-5.3 to -2.8) treated bleeding events/year, a reduction of 83.8% from baseline. The mean change in infusions/year was 134 (-144 to -124), a decrease of 98.6% from baseline. Post-infusion was defined as starting after week 4.
Safety
Annualized bleed rate (ABR, left panel) and number of FVIII infusions/year (right panel) for the rollover population (n = 112) in the GENEr8-1 trial. For ABR, the mean (95% CI) change was a decrease of 4.1 (-5.3 to -2.8) treated bleeding events/year, a reduction of 83.8% from baseline. The mean change in infusions/year was 134 (-144 to -124), a decrease of 98.6% from baseline. Post-infusion was defined as starting after week 4.

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Supported by educational grants from Bayer, BioMarin, Freeline Therapeutics Limited, Pfizer Inc., Shire, Spark Therapeutics, and uniQure, Inc.

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