FDA Approves Hemlibra to Treat Hemophilia A Without Factor VIII Inhibitors

The U.S. Food and Drug administration (FDA) has approved Genentech’s Hemlibra (emicizumab-kxwh) for routine prophylactic (preventive) treatment of patients with hemophilia A without factor VIII inhibitors.

Hemlibra is now the sole available prophylactic treatment for patients with hemophilia A with and without factor VIII inhibitors that can be given through an injection under the skin (subcutaneously), as well as at multiple dosing options (once a week, once every two weeks or once every four weeks).

The approval comes on the heels of positive data from two major clinical trials — the Phase 3 HAVEN 3 (NCT02847637) and HAVEN 4 (NCT03020160) studies — in which prophylactic treatment using Hemlibra was associated with a significant decrease in treated bleeds compared to no prophylactic treatment.

Following those results, the FDA approved Hemlibra for prophylactic treatment of both adults and children, including newborns.

For a long time, patients with hemophilia A without factor VIII inhibitors were dependent on intravenous infusions multiple times a week. Despite these regimens, patients still experienced bleeds, which underscored the need for more treatment options.

“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 first time in nearly 20 years. Hemlibra can reduce bleeds, and it offers a new subcutaneous administration once weekly, every two weeks or every four weeks,” hematologist Michael Callaghan, MD, Children’s Hospital of Michigan, said in a press release.

“Hemlibra is now the only FDA-approved medicine for people with hemophilia A with and without factor VIII inhibitors, based on the efficacy and safety profile demonstrated across four pivotal studies. We want to thank the hemophilia community for their partnership in helping us bring this new option to everyone living with hemophilia A,” added Sandra Horning, MD, chief medical officer and head of global product development at Genentech.

In the HAVEN 3 study, researchers recruited adults and adolescents who were 12 or older with hemophilia A without factor VIII inhibitors. Participants received Hemlibra, as preventive treatment, either once a week or once every two weeks.

Patients treated once a week experienced a 96% reduction, and patients treated once every two weeks experienced a 97% reduction, in treated bleeds compared to patients who did not receive prophylactic treatment.

Additionally, Hemlibra is the first therapeutic to significantly decrease the number of treated bleeds compared to previous factor VIII prophylaxis. In fact, treatment with Hemlibra was associated with a 68% reduction in treated bleeds compared to individuals who previously received factor VIII prophylaxis and switched to Hemlibra treatment.

In the HAVEN 4 study, results indicated that Hemlibra prophylaxis every four weeks led to clinically meaningful control of bleeding in adults and adolescents (12 or older) with hemophilia A with factor VIII inhibitors and without factor VIII inhibitors.

Hemlibra is now available the U.S. for patients with hemophilia A without factor VIII inhibitors.