Data from Roche’s phase 3 HAVEN 6 study show clinically meaningful bleed control, with no new safety signals observed.
Roche announced the primary analysis of the phase 3 HAVEN 6 study, which demonstrated that emicizumab (Hemlibra) continued to demonstrate effective bleed control and a favorable safety profile in individuals with moderate or mild hemophilia A, without VIII inhibitors.
The findings were presented at the 30th annual international Society on Thrombosis and Hemostasis (ISTH) Annual Congress in London, United Kingdom. Roche plans to use the data in support of a submission to the European Medicines Agency to update the label for emicizumab to include individuals with non-severe hemophilia A.
HAVEN 6 is a multicenter, open-label, phase 3, single-arm study that evaluated the efficacy, pharmacodynamics, pharmacokinetics, and safety of emicizumab in individuals with moderate or mild hemophilia A without factor VIII inhibitors.
The primary analysis included data from 72 individuals, 3 of whom were female, who warranted prophylaxis. Of the individuals, 21 had mild hemophilia A, and 51 had moderate at a median follow-up of approximately 55.6 weeks.
At baseline, 37 individuals were receiving factor VIII prophylactic treatment, and 24 had target joints.
These data showed that emicizumab maintained low treated bleed rates across the study period, with 66.7% of individuals experiencing no bleeds that required treatment, 81.9% experiencing no spontaneous bleeds that required treatment, and 88.9% experiencing no joint bleeds that needed treatment.
Additionally, model-based annualized bleed rates remained low throughout the evaluation period at 0.9.
Furthermore, the results showed that emicizumab’s safety profile was consistent with findings across various subpopulations of individuals with hemophilia A, from previous HAVEN and STASEY studies, with no new safety signals.
The most common adverse event (AE) was injection site reactions (ISRs). Fifteen individuals reported an emicizumab-related AE, which was majorly local ISRs. However, 1 individual did experience a grade 1 thromboembolic event, unrelated to emicizumab.
There were no cases of thrombotic microangiopathy or deaths, reinforcing emicizumab’s safety profile.
Additionally, data from the CHESS II and CHESS PAEDs studies were also presented at ISTH 2022. The data demonstrated that most individuals with mild or moderate hemophilia A and more than half of children with moderate hemophilia A may not receive preventative treatments.
As a result, this can worsen clinical burden, as more than 30% of adults and approximately 40% of children with moderate hemophilia A who were not taking preventative treatment in the study experienced at least 3 bleeds a year, according to a statement from the company.
Emicizumab is approved to treat individuals with hemophilia A with factor VIII inhibitors in more than 110 countries and for individuals without VIII inhibitors in more than 95 countries, including Japan and the United States, for all severities of hemophilia A. In the European Union, it is only approved for severe hemophilia A.
Emicizumab has been studied in 1 of the largest clinical trial programs in individuals with hemophilia A with and without factor VIII inhibitors, including 8 phase 3 studies, according to the statement.
New data from phase III HAVEN 6 study reinforce favorable safety and efficacy profile of Roche’s Hemlibra in people with moderate or mild hemophilia A. Roche. News release. July 11, 2022. Accessed July 13, 2022. https://www.roche.com/media/releases/med-cor-2022-07-11