FDA OKs Hemlibra for Hemophilia A Without Factor VIII Inhibitors

Officials with the FDA have approved emicizumab-kxwh (Hemlibra, Genentech) for routine prophylaxis to prevent or reduce frequency of bleeding episodes in adults and children, ages newborn and older, with hemophilia A without factor VIII inhibitors, according to a press release.

Emicizumab-kxwh is now the only prophylactic treatment approved for individuals with hemophilia A with and without factor VIII inhibitors, Genentech announced. The treatment can be administered subcutaneously and at multiple dosing options.

The FDA initially approved emicizumab-kxwh in November 2017 for adults and children with hemophilia A with factor VIII inhibitors.

The latest approval is based on the pivotal phase 3 HAVEN 3 and HAVEN 4 clinical trials, part of the largest clinical program in hemophilia A. The HAVEN 3 study, which included 152 participants, evaluated emicizumab-kxwh prophylaxis administered every week or 2 weeks in patients aged 12 years or older with hemophilia A without factor VIII inhibitors. In the HAVEN 4 study, emicizumab-kxwh was administered every 4 weeks.

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