New Data Support Hemlibra Efficacy Across Dosing Schedules in Hemophilia A with Factor VIII Inhibitors

Updated data from the HAVEN 2 study showed that the majority of children treated with emiciziumab-kxwh (Hemlibra, Genentech) experienced sustained bleed control.

Treatment with emiciziumab-kxwh (Hemlibra, Genentech) demonstrated sustained bleed control across 3 different dosing schedules in the majority of children with hemophilia A with factor VIII inhibitors, according to updated data from the phase 3 HAVEN 2 study.

Results from the study were presented at the 60th American Society of Hematology (ASH) Annual Meeting.

The HAVEN 2 study evaluated emiciziumab-kxwh prophylaxis in children younger than 12 years of age with hemophilia A with factor VIII inhibitors, including longer follow-up for once-weekly dosing and less frequent dosing schedules. The primary analysis included 85 children and the intra-patient comparison included 18 patients from the once-weekly cohort previously treated with bypassing agents (BPAs), either as prophylaxis or on-demand.

Emiciziumab-kxwh is designed to bring together factor IXa and factor X, proteins required to activate the natural coagulation cascade and restore the blood clotting process for patients with hemophilia A, according to the press release.

Based on the new data from a median of 11 additional months, 76.9% (95% CI: 64.8; 86.5) of children treated with emiciziumab-kxwh once weekly experienced 0 treated bleeds. Additionally, a prospective intra-patient comparison showed that once-weekly dosing demonstrated a 99% reduction in treated bleeds compared with prior treatment with BPAs as prophylaxis or on-demand. New data demonstrated clinically meaningful bleed control at dosing schedules of 2 weeks and 4 weeks, with 90% (95% CI: 55.5; 99.7) and 60% (95% CI: 26.2; 87.8) of children with factor VIII inhibitors experiencing 0 treated bleeds, respectively.

These data reinforce emiciziumab-kxwh’s ability to provide sustained and effective bleed control with the option for different treatment schedules, according to Genentech.

“The updated analysis from the HAVEN 2 study supports the potential of Hemlibra to control bleeds at less frequent subcutaneous dosing, providing parents and their children more flexibility to choose a treatment schedule that is right for them,” Sandra Horning, MD, chief medical officer and head of Global Product Development at Genentech, said in a statement.

In October 2018, the FDA also approved emiciziumab-kxwh for use in patients of all ages with hemophilia A without factor VIII inhibitors. It is currently the only approved treatment for those with and without factor VIII inhibitors that can be administered subcutaneously and at multiple dosing schedules, according to the release.

Reference

Genentech’s Hemlibra (emiciziumab-kxwh) Provided Sustained Bleed Control in the Largets Pivotal Study to Date of Children With Hemophilia Factor VIII Inhibitors [news release]. Genentech’s website. https://www.gene.com/media/press-releases/14768/2018-12-03/genentechs-hemlibra-emicizumab-kxwh-prov. Accessed December 12, 2018.