Roche's Hemlibra provided sustained bleed control in the largest pivotal study to date of children with haemophilia A with factor VIII inhibitors

F. Hoffmann-La Roche Ltd / Roche's Hemlibra provided sustained bleed control in the largest pivotal study to date of children with haemophilia A with factor VIII inhibitors . Processed and transmitted by West Corporation. The issuer is solely responsible for the content of this announcement.

• Nearly 77% of children receiving Hemlibra once weekly experienced zero treated bleeds

Hemlibra once weekly reduced treated bleeds by 99% compared to prior bypassing agents in a prospective intra-patient comparison

Hemlibra every two weeks and every four weeks also showed clinically meaningful control of bleeding

             
Basel, 3 December 2018 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced data from the primary analysis of the phase III HAVEN 2 study evaluating Hemlibra (emicizumab) prophylaxis in children younger than 12 years of age with haemophilia A with factor VIII inhibitors, including longer follow-up for once-weekly dosing and new data for less frequent dosing schedules (every two weeks or every four weeks). These data from the largest pivotal study in children with haemophilia A with factor VIII inhibitors were presented at the 60th American Society of Hematology (ASH) Annual Meeting.

"Children with inhibitors are at increased risk of life-threatening bleeds and may experience frequent, repeated bleeding into joints," said Guy Young, MD, Director of Hemostasis and Thrombosis Center, Children's Hospital Los Angeles, and Professor of Pediatrics, University of Southern California Keck School of Medicine, Los Angeles, California. "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."

In updated results from the HAVEN 2 study with a median of 11 additional months of data, 76.9% (95% CI: 64.8; 86.5) of children with haemophilia A with factor VIII inhibitors treated with Hemlibra once weekly (n=65) experienced zero treated bleeds. Importantly, once-weekly Hemlibra showed a 99% (95% CI: 97.7; 99.4) reduction in treated bleeds compared to prior treatment with bypassing agents (BPAs) as prophylaxis (n=15) or on-demand (n=3) in a prospective intra-patient comparison. New data also showed that 90% (95% CI: 55.5; 99.7) of children with factor VIII inhibitors receiving Hemlibra every two weeks (n=10) and 60% (95% CI: 26.2; 87.8) of children receiving Hemlibra every four weeks (n=10) experienced zero treated bleeds, demonstrating clinically meaningful bleed control at both dosing schedules. No cases of thrombotic microangiopathy (TMA) or thrombotic events occurred. The most common adverse events (AEs) in the HAVEN 2 study primary analysis were consistent with those previously observed in the interim analyses.