A novel, von Willebrand factor (VWF)-independent, extended half-life factor VIII (FVIII) therapy may allow patients with hemophilia A to pursue once-weekly, or less frequent, dosing rather than prophylactic dosing, according to preliminary study findings published in Haemophilia.

Currently, weekly dosing remains out of reach for patients with severe hemophilia A because of the speed with which FVIII degrades, both when carried by VWF and in the absence of VWF. The authors of this study suggested that BIVV001 (recombinant factor VIII Fc fusion protein [rFVIIIFc]-VWF-XTEN) may present a solution to this problem. The therapy functions independently of VWF, taking advantage of both the Fc properties of rFVIIIFc and the XTEN polypeptides to potentially extend the half-life of FVIII.

In this phase 1/2a clinical study (ClinicalTrials.gov Identifier: NCT03205163), patients received 1 dose each of full-length rFVIII and BIVV001 separated by a washout period. Patients were assessed for presence of inhibitors at 14 and 28 days after BIVV001-dosing as well as FVIII activity throughout the study. The primary endpoints of the study were safety and tolerability of 1 dose of BIVV001; secondary endpoints included assessment of pharmacokinetic (PK) parameters.

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The patients in this study comprised a low-dose cohort. Additional patients will be enrolled into a high-dose cohort following data review by the sponsor of the study and the Data Safety Monitoring Committee.

“This study will provide the first BIVV001 safety and PK data in patients with hemophilia A,” the authors concluded.

Disclosure: Multiple authors declare affiliations with the pharmaceutical industry. For a full list of disclosures please refer to the original study.

Reference

  1. Shapiro A, Quon D, Staber J, et al. BIVV001 – a novel, weekly dosing, VWF-independent, extended half-life FVIII therapy: First-in-human safety, tolerability, and pharmacokinetics [Abstract M-LBMED01-004 (858)]. Haemophilia. 2018;24(suppl. 5):210.