Commitment to Advancing Care for People with Hemophilia A Demonstrated with Largest and Longest, Ongoing Clinical Development Program for any Gene Therapy in Hemophilia A

European Commission Approval for Valoctocogene Roxaparvovec Expected Q3 2022

SAN RAFAEL, Calif., July 11, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today announced that it presented four oral presentations and two poster presentations on valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A, at the International Society on Thrombosis and Haemostasis (ISTH) 2022 World Congress from July 9-12 in London.

"With each passing year, the breadth of data supporting valoctocogene roxaparvovec increasingly demonstrate the positive impact gene therapy treatment may provide people with severe hemophilia A.  These presentations at ISTH offer supportive evidence of long-term hemostatic efficacy, consistent safety results in clinical studies, efficacy from our pivotal study that is consistent with propensity scoring, the clearance of the vector, and on health-related quality of life over six years," said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin.  "We look forward to an anticipated approval of valoctocogene roxaparvovec in Europe and providing a therapy that could represent an important and valuable treatment choice for patients with severe Hemophilia A by offering the potential to reduce both the burden of the disease as well as the burden of treatment."

"BioMarin continues to increase and share important data about investigational valoctocogene roxaparvovec that may be useful for patients and physicians to evaluate therapeutic options based on an individual's unique circumstances," said one of the presenters Professor Johnny Mahlangu, a study investigator and Professor in Haematology and Head of School of Pathology in the Faculty of Health Sciences of the University of the Witwatersrand in Johannesburg, South Africa. 

Presentation of these data at ISTH follows the Committee for Medicinal Products for Human Use (CHMP) adopting a positive opinion recommending conditional marketing authorization (CMA) for valoctocogene roxaparvovec for adults with severe hemophilia A. Valoctocogene roxaparvovec is the first gene therapy to be recommended for approval in Europe for hemophilia A.  A final approval decision, typically consistent with the CHMP recommendation, is expected from the European Commission in Q3 2022. 

BioMarin's presentations at ISTH include:

Oral Presentations

Comparative effectiveness of valoctocogene roxaparvovec and prophylactic factor VIII replacement estimated through propensity scoring

Anthony J. Hatswell, PhD, Director and Analyst, Delta Hat Limited, Nottingham, UK

Sunday July 10, 2022, 3:45 PM - 4:00 PM BST

Relationship between transgene-produced FVIII and bleeding rates 2 years after gene transfer with valoctocogene roxaparvovec: Results from GENEr8-1

Professor Johnny Mahlangu, Professor of Haematology and Head of School of Pathology in the Faculty of Health Sciences of the University of the Witwatersrand in Johannesburg, South Africa. 

Sunday July 10, 2022, 3:15 PM - 3:30 PM BST

Hemostatic results for up to 6 years following treatment with valoctocogene roxaparvovec, an AAV5-hFVIII-SQ gene therapy for severe hemophilia A

Professor Michael Laffan, faculty of Medicine, Department of Immunology and Inflammation at Imperial College London, Director of the Hammersmith Hospital Haemophilia Centre

Sunday July 10, 2022, 3:00 PM - 3:15 PM BST

Innate and Adaptive Immune Responses to Adeno-associated viral Gene Therapy in the Severe Hemophilia A Dog Model

Paul Batty, MBBS, PhD, Associate Professor at University College London and an Honorary Consultant in Haemostasis and Thrombosis at the Katharine Dormandy Haemophilia and Thrombosis Centre (Royal Free Hospital, London, UK)

Saturday, July 9, 2022, 2:00 PM – 2:15 PM BST

Posters

Comparability of bleeding outcomes by prophylactic FVIII replacement intensity: A post hoc analysis of a noninterventional study of men with severe hemophilia A

Charlotte Camp, MSc, Associate Director, Health Economics and Outcomes Research, BioMarin

Monday July 11, 2022, 6:30 PM - 7:30 PM BST

Blood Biodistribution and Vector Shedding of Valoctocogene Roxaparvovec in People with Severe Hemophilia A: Results from the Phase 3 GENEr8-1 Trial

Suresh Agarwal, PhD, MS, RPh, Director, Clinical Pharmacology, BioMarin

Sunday July 10, 2022, 6:30 PM - 7:30 PM

About Hemophilia A

People living with hemophilia A lack sufficient functioning Factor VIII protein to help their blood clot and are at risk for painful and/or potentially life-threatening bleeds from even modest injuries. Additionally, people with the most severe form of hemophilia A (FVIII levels