BioMarin Pharmaceutical Inc. (BMRN), in an update on phase 1/2 study of valoctocogene roxaparvovec, an investigational gene therapy treatment for adults with severe hemophilia A, said Wednesday that the safety profile of valoctocogene roxaparvovec in the study remains consistent with previously reported data with no delayed-onset treatment related adverse events.
The company noted that the latest data update in the ongoing study represents the longest duration of clinical experience for any gene therapy in hemophilia A and demonstrates hemostatic control with valoctocogene roxaparvovec out to five years in the majority of patients in this study.
Five-year and four-year post-treatment follow-up of the 6e13 vg/kg and 4e13 vg/kg cohorts, respectively, showed a sustained treatment benefit of valoctocogene roxaparvovec. All participants in both cohorts remain off prophylactic Factor VIII treatment.
Mean cumulative annualized bleed rates (ABR) remain less than one in the 6e13 vg/kg cohort and substantially below pre-treatment baseline levels; the mean ABR in year five for the 6e13 vg/kg cohort was 0.7 with an ABR reduction of 95% and Factor VIII use reduction of 96% through five years, compared to pre-infusion.
The mean ABR in year four for the 4e13 vg/kg cohort was 1.7 with a mean cumulative ABR reduction of 92% and Factor VIII use reduction of 95% through four years, compared to pre-infusion.
Factor VIII activity levels declined commensurate with the most recent years' observations and continue to remain in a range to provide hemostatic efficacy.
According to the company, no participants developed inhibitors to Factor VIII, and no participants withdrew from the study. No participants have developed thrombotic events.
The most common adverse events associated with valoctocogene roxaparvovec occurred early after a single infusion and included short-lived infusion-associated reactions and transient, asymptomatic, and mild to moderate rise in the levels of certain proteins and enzymes measured in liver function tests with no long-lasting clinical sequelae.
In Europe, BioMarin plans to submit a Marketing Authorization Application for valoctocogene roxaparvovec for the treatment of severe hemophilia A with one-year results from the Phase 3 GENEr8-1 study to the European Medicines Agency in June 2021 based on positive feedback from EMA earlier this year.
In the United States, BioMarin plans to submit two-year follow-up safety and efficacy data on all study participants from the GENEr8-1 study in response to FDA's request for these data to support their benefit-risk assessment of valoctocogene roxaparvovec.
BioMarin target a Biologics License Application submission in the second quarter of 2022 assuming favorable study results, followed by an expected six-month review procedure by the FDA.
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