Roche's Genentech has presented a primary analysis at the American Society of Hematology conference of a Phase III Haven 2 study evaluating Hemlibra (emicizumab-kxwh) prophylaxis in haemophilia A patients under 12 years of age with factor VIII inhibitors, including longer follow-up for once-weekly dosing and new data for less frequent dosing schedules.

Hemlibra was given priority review in the US back in June after Roche released interim results from the trial showing that after a median of twelve weeks of treatment, prophylactic treatment with the drug led to a clinically meaningful reduction in the number of bleeds over time.

Updated results now show that 76.9% of children receiving Hemlibra once weekly experienced no treated bleeds, while 23.1% experienced one to three treated bleeds. Furthermore, once-weekly dosing also reduced treated bleeds by 99% compared to prior bypassing agents in the prospective intra-patient comparison.

Roche said the new data also shows that 90% of patients who received Hemlibra every two weeks and 60% of those treated every four weeks experienced no treated bleeds, "demonstrating clinically meaningful bleed control at both dosing schedules."

"Children with inhibitors are at increased risk of life-threatening bleeds and may experience frequent, repeated bleeding into joints," said Guy Young, 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."

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