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Hemlibra shows "clinically meaningful" bleed control in paediatric haemophilia A patients

Published on 04/12/18 at 12:14pm

In another reveal at the American Society of Hematology (ASH) Annual Meeting 2018, Roche unveiled new Phase 3 findings for Hemlibra (emicizumab) as a prophylactic in haemophilia A patients with factor VIII inhibitors younger than 12 years of age.

The data, drawn from the largest pivotal study of this patient population, showed that 76.9% of participants treated once weekly experienced zero treated bleeds over the course of the study period. The drug also delivered a 99% reduction in treated bleeds compared to prior treatment with bypassing agents as a preventative measure or on-demand, according to a prospective intra-patient comparison.

Additionally, 90% of those with factor VIII inhibitors receiving Hemlibra fortnightly saw zero treated bleeds, as did 60% receiving it monthly.

 “Children with inhibitors are at increased risk of life-threatening bleeds and may experience frequent, repeated bleeding into joints,” said Dr Guy Young, Director of Hemostasis and Thrombosis Center at the Children’s Hospital Los Angeles, and Professor of Pediatrics at the University of Southern California Keck School of Medicine. “These updated data from HAVEN 2 showed that the majority of children with haemophilia A with factor VIII inhibitors treated with emicizumab had zero treated bleeds across three different dosing schedules, reinforcing the ability of this medicine to provide sustained, effective bleed control.”

Roche’s Chief Medical Officer and Head of Global Product Development, Dr Sandra Horning, also commented: “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. Many children with haemophilia A with factor VIII inhibitors have already experienced the benefits of Hemlibra, and with these new positive data, we are confident that this treatment will continue to make a meaningful difference in their lives.”

Matt Fellows

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