BioMarin Pharmaceutical Inc. announced that the Company will be presenting data in several presentations on valoctocogene roxaparvovec, an investigational gene therapy for the treatment of adults with severe hemophilia A at the 15th Annual Virtual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) February 2-4. Notably, there will be an oral presentation of data from the two-year analysis of an ongoing global Phase 3 study, GENEr8-1, the largest Phase 3 gene therapy study in severe hemophilia A with 134 participants and the safety data of the overall program.
On Friday February 4 at 17:10 Central European Time , Prof. Johnny Mahlangu, Professor in Haematology and Head of School of Pathology in the Faculty of Health Sciences of the University of the Witwatersrand and the National Health Laboratory Service in Johannesburg, South Africa, will close the Latest Clinical Trial Results session presenting “Efficacy and safety of valoctocogene roxaparvovec gene transfer for severe haemophilia A: Results from the GENEr8-1 two-year analysis.” The entire session consisting of six presentations will be chaired by EAHAD President, Prof. Flora Peyvandi and will cover the latest developments in the field of hematology.
“We are looking forward to sharing these transformational data from the largest and longest development program for gene therapy in severe hemophilia A at EAHAD,” said Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin. “We are committed to helping the community understand the safety and efficacy of this novel investigational treatment.”
The 15th Congress of the European Association for Haemophilia and Allied Disorders is taking place from February 2 – 4, 2022. Under the direction of the Organizing Committee, the EAHAD 2022 Virtual Congress aims to continue being a locus for the latest innovations and advances in the field of hemophilia and bleeding disorders.
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 <1%) often experience painful, spontaneous bleeds into their muscles or joints. Individuals with the most severe form of hemophilia A make up approximately 50 percent of the hemophilia A population. People with hemophilia A with moderate (FVIII 1-5%) or mild (FVIII 5-40%) disease show a much-reduced propensity to bleed. The standard of care for individuals with severe hemophilia A is a prophylactic regimen of replacement Factor VIII infusions administered intravenously up to two to three times per week or 100 to 150 infusions per year. Despite these regimens, many people continue to experience breakthrough bleeds, resulting in progressive and debilitating joint damage, which can have a major impact on their quality of life.