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BioMarin Provides Highlights of 4 Years of Clinical Data from Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A

SAN RAFAEL, Calif., May 31, 2020 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today an update to its previously reported results of an open-label Phase 1/2 study of valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A. A The data have been submitted as a late-breaking abstract to the World Federation of Hemophilia (WFH) Virtual Summit to be held June 14-19, 2020.
BioMarin Provides Highlights of 4 Years of Clinical Data from Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec Gene Therapy for Severe Hemophilia A
The four-year update for the 6e13 vg/kg and three-year update for the 4e13 vg/kg cohorts demonstrated that all subjects in both cohorts remain off prophylactic Factor VIII treatment since receiving their single dose of valoctocogene roxaparvovec. Cumulative mean annualized bleed rates (ABR) remain less than one (1) in both cohorts and below pre-treatment baseline levels.A  The mean ABR in year four for the 6e13 vg/kg cohort was 1.3, and the mean ABR in year three for the 4e13 vg/kg cohort was 0.5.A  Over the past year, six of the seven participants in the 6e13 vg/kg cohort and five of the six participants in the 4e13 vg/kg cohort remain free of spontaneous bleeds.A  Factor VIII activity levels declined commensurate with the most recent years' observations and remain in a range to provide hemostatic efficacy.A  "It's been a privilege to participate in this pioneering research and to observe how the patients on the study have done so much to improve our understanding of gene therapy research for hemophilia A.A  This additional data is an important step toward a potential first treatment of its kind for this devastating disease," said Professor John Pasi, M.B., Ch.B., Ph.D., from Barts and the London School of Medicine and Dentistry; chief investigator for the valoctocogene roxaparvovec Phase 1/2 study, and a principal investigator for the Phase 3 study.A  "Each year of data increases our knowledge of safety and efficacy and contributes to the growing body of scientific data on gene therapies in general and hemophilia A in particular.""BioMarin is proud to have advanced the community's knowledge of the potential for gene therapy to transform lives, and we are grateful for the support of the bleeding disorders community in this endeavor. In just over four years since starting clinical trials in patients, we've submitted applications for marketing authorizations globally, and we continue to contribute to the growing body of scientific data in gene therapy for hemophilia A with five studies underway," said Hank Fuchs, M.D., President, Worldwide Research and Development at BioMarin.A  "We continue to move forward with health authorities to make this treatment available for people with severe hemophilia A.A  A We are committed to pioneering this field and advancing the standard of care for patients." A 
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