Roche announced that the US Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to Hemlibra (emicizumab-kxwh) for people with haemophilia A without factor VIII inhibitors. Breakthrough Therapy Designation is designed to accelerate the development and review of medicines intended to treat a serious condition with preliminary evidence that indicates they may demonstrate a substantial improvement over existing therapies.

“Hemlibra is the first medicine to show superior efficacy compared to factor VIII prophylaxis, the standard of care for people with haemophilia A without inhibitors, in an intra-patient comparison,” said Sandra Horning, MD, Roche’s chief medical officer and head of global product development. “We look forward to working with health authorities to make Hemlibra available to people without inhibitors as soon as possible, and we are excited to share this news with the community as we join in celebrating World Hemophilia Day.”

This designation is based on data from the phase III HAVEN 3 study in people 12 years or older with haemophilia A without inhibitors. In the study, Hemlibra prophylaxis dosed subcutaneously every week or every two weeks showed a statistically significant and clinically meaningful reduction in treated bleeds compared to no prophylaxis. In an intra-patient comparison, once-weekly Hemlibra prophylaxis was superior to prior factor VIII prophylaxis as demonstrated by a statistically significant and clinically meaningful reduction in treated bleeds. The most common adverse events with Hemlibra were injection site reactions, and no new safety signals were observed. No thrombotic microangiopathy or thrombotic events occurred in this study.

Hemlibra was granted its first Breakthrough Therapy Designation in September 2015 and was approved by the FDA in November 2017 for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with haemophilia A with factor VIII inhibitors based on results from the HAVEN 1 and HAVEN 2 studies. Hemlibra was also recently approved by regulatory authorities in other countries around the world, including by the European Commission in February 2018 for routine prophylaxis of bleeding episodes in people with haemophilia A with factor VIII inhibitors.

The Hemlibra development programme reflects Roche’s commitment to address clinical unmet needs in the haemophilia A community. Roche and Genentech are proud to support the World Federation of Hemophilia and the global bleeding disorders community as sponsors of World Hemophilia Day.

HAVEN 3 is a randomised, multicentre, open-label, phase III study evaluating the efficacy, safety and pharmacokinetics of Hemlibra prophylaxis versus no prophylaxis (episodic/on-demand factor VIII treatment) in people with haemophilia A without inhibitors to factor VIII. The study included 152 patients with haemophilia A (12 years of age or older) who were previously treated with factor VIII therapy either on-demand or for prophylaxis. Patients previously treated with on-demand factor VIII were randomised in a 2:2:1 fashion to receive subcutaneous Hemlibra prophylaxis at 3 mg/kg/wk for 4 weeks, followed by 1.5 mg/kg/wk until the end of study (Arm A), subcutaneous Hemlibra prophylaxis at 3 mg/kg/wk for 4 weeks, followed by 3 mg/kg/2wks until the end of study (Arm B), or no prophylaxis (Arm C). Patients previously treated with factor VIII prophylaxis received subcutaneous Hemlibra prophylaxis at 3 mg/kg/wk for 4 weeks, followed by 1.5 mg/kg/wk until the end of study (Arm D). Episodic treatment of breakthrough bleeds with factor VIII therapy was allowed per protocol.

Hemlibra is a bispecific factor IXa- and factor X-directed antibody. It 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 people with haemophilia A. Hemlibra is a prophylactic (preventative) treatment that can be administered by an injection of a ready-to-use solution under the skin (subcutaneously) once-weekly.

The clinical development programme is assessing the safety and efficacy of Hemlibra and its potential to help overcome current clinical challenges: the short-lasting effects of existing treatments, the development of factor VIII inhibitors and the need for frequent venous access. Hemlibra was created by Chugai Pharmaceutical Co., Ltd. and is being co-developed by Chugai, Roche and Genentech. It is marketed in the United States as Hemlibra (emicizumab-kxwh) for people with haemophilia A with factor VIII inhibitors, with kxwh as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the US Food and Drug Administration.