Business
BioMarin Announces Oral Presentation at International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress with 5 Years of Clinical Data from Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec in Adults with Severe Hemophilia A, Demon...
BioMarin Announces Oral Presentation at International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress with 5 Years of Clinical Data from
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"BioMarin Announces Oral Presentation at International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress with 5 Years of Clinical Data from Ongoing Phase 1/2 Study of Valoctocogene Roxaparvovec in Adults with Severe Hemophilia A, Demonstrating Continued, Durable Clinical Benefit \n Sustained Clinical Hemostatic Efficacy Observed in Study Participants with Longest Duration of Clinical Experience for any Gene Therapy in Hemophilia A\n Phase 1/2 and Subset of Pivotal GENEr8-1 Studies Demonstrate Consistent Bleed Control following Treatment with Valoctocogene Roxaparvovec\n All Study Participants in 6e13 vg/kg and 4e13 vg/kg Dose Cohorts Remain off Factor VIII Prophylactic Therapy\n\n\nSAN RAFAEL, Calif., July 21, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today new data for valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A, from its open-label Phase 1/2 study during an oral presentation at the International Society on Thrombosis and Haemostasis (ISTH) 2021 Virtual Congress. \n\n \n \n \n \n \n \n\n \nFive-year and four-year post-treatment follow-up of the 6e13 vg/kg and 4e13 vg/kg cohorts, respectively, shows a sustained treatment benefit of valoctocogene roxaparvovec. All participants in both cohorts remain off prophylactic Factor VIII treatment. \nAnnualized Bleed Rate\nThe 6e13 vg/kg dose cohort of the Phase 1/2 study (N=7), with a mean follow-up of 266.1 weeks (5.1 years), showed that a single dose of valoctocogene roxaparvovec after week 4 reduced mean ABR by 95% from 16.3 (median 16.5) at baseline to 0.8 (median 0.0) bleeding episodes per year among the 6 participants previously treated with FVIII prophylaxis. In year 5, 86% (6 of 7) of study participants in the 6e13 vg/kg dose cohort had no treated bleeds.\nThe 4e13 vg/kg dose cohort of the Phase 1/2 study (N=6), with a mean follow-up of 218.6 weeks (4.2 years) showed that a single dose of valoctocogene roxaparvovec reduced mean ABR by 92% from 12.2 (median 8.0) at baseline to 1.0 (median 0.5) bleeding episodes per year. In year 4, 50% (3/6) of study participants in the 4e13 vg/kg dose cohort had no treated bleeds.\nFactor VIII Utilization\nIn the 6e13 vg/kg dose cohort after week 4, the mean annualized Factor VIII utilization was reduced by 96% from 135.6 (median 136.5) t...