Relationship Between Transgene-Produced FVIII and Bleeding Rates 2 Years After Gene Transfer With Valoctocogene Roxaparvovec: Results From Gener8-1
Highlights From the 30th Congress of ISTH

Relationship Between Transgene-Produced FVIII and Bleeding Rates 2 Years After Gene Transfer With Valoctocogene Roxaparvovec: Results From Gener8-1

Presented by: Johnny Mahlangu, BSc, MBBCh, MMed, FCPath, Faculty of the Health Sciences-University of the Witwatersrand and National Health Laboratory Service, Johannesburg, South Africa

J. Mahlangu1, H. Chambost2, S. Chou3, A. Dunn4, A. von Drygalski5, R. Kaczmarek6, G. Kenet7, M. Laffan8, A. Leavitt9, B. Madan10, J. Mason11, J. Oldenburg12, M. Ozelo13, F. Peyvandi14, D. Quon15, M. Reding16, S. Shapiro17, H. Yu18, T. Robinson18, S. Pipe19

1University of the Witwatersrand, National Health Laboratory Service and Hemophilia Comprehensive Care Center, Charlotte Maxeke Johannesburg Academic Hospital, Johannesburg, Gauteng, South Africa
2APHM, Department of Pediatric Hematology Oncology, La Timone Children’s Hospital & Aix Marseille Univ, INSERM, INRA, C2VN, Marseille, Provence-Alpes-Cote d'Azur, France
3Division of Hematology, Department of Internal Medicine, National Taiwan University Hospital, Taipei, Keelung, Taiwan (Republic of China)
4Nationwide Children's Hospital Division of Hematology, Oncology, and Bone Marrow Transplant and The Ohio State University College of Medicine, Columbus, Ohio, United States
5Division of Hematology/Oncology, Department of Medicine, University of California San Diego, San Diego, California, United States
6Indiana University School of Medicine, IUPUI-Wells Center for Pediatric Research, and Hirszfeld Institute of Immunology and Experimental Therapy, Indianapolis, Indiana, United States
7Sheba medical center& Tel Aviv University, Tel Aviv, Tel Aviv, Israel
8Centre for Haematology, Imperial College London, London, England, United Kingdom
9University of California San Francisco, San Francisco, California, United States
10Guy's & St. Thomas' NHS Foundation Trust, London, England, United Kingdom
11Queensland Haemophilia Centre, Royal Brisbane and Women's Hospital, and University of Queensland, Brisbane, Queensland, Australia
12Institute of Experimental Hematology and Transfusion Medicine and Centre for Rare Diseases, Universitätsklinikum Bonn, Bonn, Nordrhein-Westfalen, Germany
13Hemocentro UNICAMP, Department of Internal Medicine, School of Medical Sciences, University of Campinas, Campinas, Sao Paulo, Brazil
14Fondazione IRCCS Ca’ Granda - Ospedale Maggiore Policlinico, UOC Medicina Generale, Angelo Bianchi Bonomi Hemophilia and Thrombosis Center and Fondazione Luigi Villa, and Department of Pathophysiology and Transplantation, Università degli Studi di Milano, Milan, Lombardia, Italy
15Orthopaedic Hemophilia Treatment Center, Los Angeles, California, United States
16Center for Bleeding and Clotting Disorders, University of Minnesota, Minneapolis, Minnesota, United States
17Oxford University Hospitals National Health Service Foundation Trust, University of Oxford, and Oxford National Institute for Health Research Biomedical Research Centre, Oxford, England, United Kingdom
18BioMarin Pharmaceutical, Inc., Novato, California, United States

19Departments of Pediatrics and Pathology, University of Michigan, Ann Arbor, Michigan, United States

Key Data Points

Valoctocogene Roxaparvovec Safety Summary for Year 2
Annualized Treated Bleeding Rate/FVIII Utilization

Results from the phase 3 GENEr8-1 study in patients with hemophilia A treated with valoctocogene roxaparvovec show a consistent reduction in treated bleeds/year and annualized FVIII utilization from year 1 to year 2 compared to baseline. Baseline values were from the 6-month prospective data collection period in the noninterventional study BMN 270-902.

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