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SAN RAFAEL, Calif., Feb. 2, 2022 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) today 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 (CET), 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.
Further demonstrating the Company's commitment to advancing the standard of care in severe hemophilia A, BioMarin also will present three posters, which will be available to meeting participants at any time during EAHAD.
Poster Title | Authors |
Health-related quality of life following valoctocogene roxaparvovec gene therapy for severe haemophilia A in the phase 3 trial GENEr8-1 | O'Mahony B, Mahlangu J, Peerlinck K, Wang JD, Lowe G, Tan C, Tran h, Khoo TL, Cockrell E, Pepperell D, Chambost H, Lopez Fernandez MF, Kazmi R, Majerus E, Skinner M, Klamroth R, Quinn J, Yu H, Wong W, Lawal A, Robinson T, Kim B |
Valoctocogene roxaparvovec gene transfer in participants with HIV | Ragni M, Majerus E, Giermasz A, Fong S, Yates B, Scheeler S, Razon L, Liu S, Yu H, Reddy D, Robinson T |
Qualitative research evaluating patient preference for haemophilia therapy | Miesbach W, Valentino L, Noone D, Forsyth K, Bullinger M, Dashiell-Aje E, Newman V, Hawes C, Hawley S, Lewis H, Latibeaudiere-Gardner D, Quinn J |
"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.
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
