Chugai Pharma announces positive results from phase III HAVEN 7 study of Hemlibra to treat infants with severe haemophilia A

Chugai Pharmaceutical Co., Ltd. announced that the primary analysis of the phase III HAVEN 7 study reinforced the efficacy and safety of Hemlibra (generic name: emicizumab) in previously untreated or minimally treated infants with severe haemophilia A without factor VIII inhibitors. Results showed that Hemlibra achieved meaningful bleed control in babies up to 12 months of age, and was well tolerated. The new data were presented at the 65th American Society of Haematology (ASH) Annual Meeting and Exposition taking place 9-12 December 2023, in San Diego, California, and included in the press programme.

“Hemlibra, which can be administered subcutaneously, is an option to reduce the treatment burden for infants who have difficulty with intravenous administration in the treatment of severe haemophilia A to prevent bleeding. In this study, Hemlibra demonstrated effective bleeding control in infants for the first time. This complements data across a wide range of ages shown in previous clinical trials and supports earlier initiation of Hemlibra treatment aimed at preventing bleeding in infants. We remain committed to building evidence including long-term data to support the safe use of this drug for those who need it,” said Dr. Osamu Okuda, Chugai’s president and CEO.

The burden of severe haemophilia A in babies and on their parents and caregivers is significant. The World Federation of Haemophilia treatment guidelines consider the standard of care in haemophilia to be regular prophylaxis initiated at a young age, as studies have shown this improves long-term outcomes, while reducing the risk of intracranial haemorrhage. However, for many babies with haemophilia A, prophylaxis is not started until after the first year of life. Hemlibra, which is already approved and being used to treat babies with haemophilia A, provides a flexible treatment option that can be administered subcutaneously from birth at different dosing frequencies for maintenance dosing.

The HAVEN 7 study is a phase III, descriptive, single-arm study, set up in collaboration with the haemophilia A community to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of subcutaneous Hemlibra in infants with severe haemophilia A without factor VIII inhibitors. These results, which included data from 55 participants, showed that at 101.9 weeks median follow-up, 54.5% of participants (n=30) did not have any bleeds that required treatment, while 16.4% (n=9) did not have any treated or untreated bleeds at all. There were no spontaneous bleeds requiring treatment in any participant, and all treated bleeds were as a result of trauma. A total of 207 bleeds occurred in 46 participants (83.6%); 87.9% of these were as a result of trauma. Model-based annualized bleeding rate (95% CI) was 0.4 (0.30-0.63) for treated bleeds. No new safety signals were observed and there were no treatment-related serious adverse events, intracranial haemorrhages or deaths reported. 3.6% of participants (n=2) tested positive for factor VIII inhibitors which may be a consequence of reduced factor VIII usage in participants treated with Hemlibra, and no participant tested positive for anti-drug antibodies. Results were consistent with positive results from the interim analysis and from previous phase III HAVEN studies.

The results of additional research on biomarkers in the HAVEN 7 study were also presented at ASH, and were supportive of the study’s primary efficacy analysis. This additional research showed that the pharmacodynamic profiles of Hemlibra in babies were consistent with those previously observed in older children and adults with haemophilia A. The data showed that Hemlibra exhibits the expected pharmacodynamic response, despite the reduced presence of the clotting factors that Hemlibra binds to in this age group.

The HAVEN 7 study results complement data from the broader, pivotal HAVEN clinical programme, providing insights into the evolution of haemophilia A in babies, and the impact of initiating preventative treatment from birth. The primary analysis is being followed by a seven year extension period.

Hemlibra is a bispecific monoclonal antibody created with Chugai’s proprietary antibody engineering technologies. The drug is designed to bind factor IXa and factor X. In doing so, Hemlibra provides the cofactor function of factor VIII in people with haemophilia A, who either lack or have impaired coagulation function of factor VIII. The product was approved by the US Food and Drug Administration (FDA) in November 2017, for the first time in the world, for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and paediatric patients with haemophilia A (congenital factor VIII deficiency) with factor VIII inhibitors. Hemlibra has been approved in more than 115 countries for congenital haemophilia A with and without factor VIII inhibitors. In Japan, it was first approved in March 2018 for congenital haemophilia A with factor VIII inhibitors, and its indication was later expanded to include congenital haemophilia A without factor VIII inhibitors, and acquired haemophilia A.

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