BioMarin’s Valoctocogene Roxaparvovec proves its ability to sustain blood factor levels.

Eighteen months of results are in and, according to data presented at the 2017 American Society of Hemophilia meeting, a 6e13 vg/kg dose of BioMarin’s valoctocogene roxaparvovec investigational gene therapy reliably sustained normal Factor VIII levels in patients’ blood after being treated for severe Hemophilia A.

BioMarin updated the efficacy of two doses of its treatment at the meeting. Of the three patients with the longest follow-up (week 48) associated with its 4e13 vg/kg dose, their Factor VIII activity levels were “in or near to the normal range with both median and mean values of 49%.” Median annualized bleed factor rates after week 4 for the 4e dose cohort were zero after patient Factor VIII activity rose above 49%.

Similarly, at 78 weeks post-infusion, the median and mean Factor VIII levels of the 6e13 vg/kg cohort were 90 and 89%, respectively. BioMarin noted the median annualized bleed and factor VIII rates for the 6e dose cohort were zero after week 4.

Hank Fuchs, BioMarin’s President of Worldwide Research and Development explained: "The confluence of new medicines and advanced treatment approaches for hemophilia has created an unprecedented opportunity to improve outcomes for patients today and in the future…We are entering a new era for the treatment of severe hemophilia and look forward to advancing an innovative therapeutic platform for our patients."