Hemlibra Receives FDA Approval for Hemophilia A Without Factor VIII Inhibitors


Emicizumab-kxwh (Hemlibra) is now the only FDA-approved treatment for hemophilia A with and 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.

Data from the HAVEN 3 trial showed that administration of emicizumab-kxwh prophylaxis every week or every 2 weeks led to a 96% and 97% reduction in treated bleeds, respectively, compared with those who received no prophylaxis. Additionally, 55.6% of patients administered emicizumab-kxwh every week and 60% who received emicizumab-kxwh every 2 weeks experienced 0 bleeds.

Furthermore, once-weekly emicizumab-kxwh prophylaxis showed superior efficacy compared with prior factor VIII prophylaxis as demonstrated by a 68% reduction in treated bleeds in an intra-patient comparison.

In the HAVEN 4 study, which included 48 patients, emicizumab-kxwh prophylaxis showed clinically meaningful control of bleeding in adults and adolescents aged 12 years or older with hemophilia A without factor VIII inhibitors. According to the findings, 56.1% of patients experienced 0 treated bleeds and 90.2% experienced 3 or fewer treated bleeds.

Emicizumab-kxwh was previously granted Breakthrough Therapy Designation and Priority Review by the FDA for this indication.

“Many preventative treatment options for people with hemophilia A without factor VIII inhibitors require intravenous infusions several times a week. Even then, people can still experience bleeds, and there has been a need for more treatment options,” Michael Callaghan, MD, hematologist at Children’s Hospital of Michigan, said in the release. “The approval of Hemlibra is an important advancement for the entire hemophilia A community, as we now have a new class of medicine for the time in nearly 20 years.”

Hemlibra was created by Chugai Pharmaceutical Co and is being co-developed by Chugai, Roche, and Genentech.


FDA Approves Genentech’s Hemlibra (Emicizumab-kxwh) for Hemophilia A Without Factor VIII Inhibitors [news release]. Genentech’s website. https://www.gene.com/media/press-releases/14749/2018-10-04/fda-approves-genentechs-hemlibra-emicizu. Accessed October 4, 2018.

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