Novel data on emicizumab-kxwh report its favorable performance among patients with hemophilia A, according to a recent press release by Genentech.

Phase 3 of the HAVEN 6 study reported that 66.7% of all participants had no overall episodes of bleeding that required additional treatment in a median follow-up of 55.6 weeks without any novel safety signals. Moreover, 81.9% and 88.9% had no spontaneous bleeds and no joint bleeds that required treatment, respectively. Annualized bleed rates were also low during this period at 0.9.

The safety profile did not change when compared to the previous HAVEN and STASEY studies, with 20.8% of the patients experiencing side effects. Local reaction in the injection site was the most frequent adverse event, occurring in 16.7% of the participants. Only 1 individual had a thromboembolic event, and there were no thrombotic microangiopathies or deaths.


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This multicenter open-label, single-arm study aimed to assess the efficacy and safety of emicizumab-kxwh in patients previously diagnosed with mild to moderate hemophilia A without factor VIII inhibitors. To obtain these results, the researchers included a total of 72 individuals, mostly (69) men, of whom 51 had the moderate presentation of the disease and 21 the mild form.

Read more about hemophilia therapies

Emicizumab-kxwh, a bispecific direct antibody for factors IXa and X joins both proteins to activate the coagulation cascade, performing a preventive action for patients with hemophilia A, the most common form of hemophilia, with up to 60% of the patients experiencing the severe presentation of this disorder.

Data also suggest that most patients with this disease do not receive prophylactic treatment, possibly translating into higher morbidity rates since 40% of children and 30% of adults with hemophilia A are thought to have at least 3 bleeding episodes per year.

This drug is currently approved in at least 110 countries for newborns, children, and adults, and its recommended dosing scheme consists of a subcutaneous injection administered every 1, 2, or 4 weeks.

Reference

New data from phase III HAVEN 6 study reinforce favorable safety and efficacy profile of Genentech’s Hemlibra (emicizumab-kxwh) in people with moderate or mild hemophilia A. News release. Genentech; July 11, 2022.