Roche Highlights Long-Term Efficacy and Safety of Hemlibra in Hemophilia A with Positive Phase III Data

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The 62nd Annual Meeting of the American Society of Hematology (ASH) was held from December 5 to 8, 2020. At this meeting, Roche presented new analytical results from a pooled three-year follow-up of 401 patients with hemophilia A (including children, adolescents, and adults) across four pivotal HAVEN studies (HAVEN 1–4). The HAVEN program represents one of the largest pivotal clinical trial programs conducted in the field of hemophilia A. The newly presented data, building upon previously observed findings, further reinforce the long-term efficacy and safety profile of Hemlibra (emicizumab) for the treatment of hemophilia A.

This analysis includes pooled data from 401 patients with hemophilia A, with and without factor VIII inhibitors, from four key HAVEN studies (HAVEN 1, n=113; HAVEN 2, n=88; HAVEN 3, n=152; HAVEN 4, n=48), with a median duration of exposure of 120.4 weeks.

Hemlibra maintained a low treated bleed rate throughout the study period, with the model-based annualized bleed rate (ABR) remaining low at 1.4 (95% CI: 1.1–1.7) across the entire evaluation period. Meanwhile, the proportion of patients with zero treated bleeds increased during each consecutive 24-week period (70.8–83.7%). Furthermore, regarding Hemlibra prophylactic treatment, 95.1% of target joints were resolved. The results indicate that the safety profile of Hemlibra was consistent with previous observations, and no new safety signals were observed during longer-term follow-up.

Furthermore, the first interim analysis of the European Haemophilia Safety Surveillance (EUHASS) database was also presented at the ASH Annual Meeting. The analysis demonstrated that the real-world safety profile of Hemlibra is consistent with that observed in clinical trials, with no new or emerging safety signals. EUHASS is a large-scale pharmacovigilance program designed to monitor the safety of therapies for hereditary bleeding disorders.

Mechanism of Action of Hemlibra

Hemlibra is a bispecific monoclonal antibody that brings together two proteins essential for activating the natural coagulation cascade and restoring the natural coagulation process—coagulation factors IXa and X—thereby restoring hemostasis in patients with hemophilia A. Clinical studies have demonstrated that Hemlibra significantly reduces bleeding episodes and improves physical function. Hemlibra remains highly effective in patients with factor VIII inhibitors (anti-FVIII antibodies).

Hemlibra has been evaluated in the largest pivotal clinical trial program in the field of hemophilia A, comprising eight Phase III studies. This program assessed the efficacy and safety of Hemlibra, as well as its potential to address current clinical challenges in hemophilia A, including the short duration of action of existing therapies, the development of factor VIII inhibitors, and the need for frequent intravenous infusions.

Data from the HAVEN studies support the approval of Hemlibra in more than 90 countries worldwide for the treatment of patients with hemophilia A with factor VIII inhibitors (based on the HAVEN 1 and HAVEN 2 studies), and its approval in more than 80 countries globally (including the United States, the European Union, and Japan) for the treatment of patients with hemophilia A without factor VIII inhibitors (based on the HAVEN 3 and HAVEN 4 studies), to prevent or reduce the frequency of bleeding events. Other studies, including HAVEN 5, HAVEN 6, HAVEN 7, HAVEN 8, and STASEY, reflect Roche’s ongoing commitment to characterizing the safety and efficacy of Hemlibra and addressing unmet medical needs in the hemophilia A community.

Since its initial approval more than three years ago, Hemlibra has been administered to a total of 8,200 patients worldwide. In the United States, Hemlibra is now the most frequently prescribed prophylactic treatment for patients with hemophilia A.

Reference source: New follow-up phase III data reinforce the long-term benefit of Roche’s Hemlibra for people with haemophilia A