Dec 5, 2017

Genentech recently reported results from the phase III HAVEN 3 study, which showed that the prophylactic use of HEMLIBRA® led to significantly reduced bleeds in adult and adolescent patients with hemophilia A, without inhibitors. The therapy was co-developed by Genentech, Chugai and Roche.

The announcement by Genentech follows closely on the US Food and Drug Administration (FDA) approval of HEMLIBRA for routine, once-weekly prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with factor VIII inhibitors – the National Hemophilia Foundation reported on this approval in November. While existing factor therapies are administered intravenously, HEMLIBRA is novel in that it is delivered subcutaneously through an injection just under the skin.

As a multicenter clinical trial, HAVEN 3 was designed to evaluate the efficacy, safety and pharmacokinetics of HEMLIBRA prophylaxis versus episodic/on-demand factor VIII treatment in people with hemophilia A without inhibitors to factor VIII. The study included 152 patients with hemophilia A (12 years of age or older) who were previously treated with factor VIII therapy either on-demand or on prophylaxis.

According to a Genentech press release, the study showed a “statistically significant and clinically meaningful” reduction in the number of treated bleeds over time in both patients receiving HEMLIBRA prophylaxis every week AND in those receiving the therapy every two weeks, compared to individuals receiving no prophylaxis. In addition, once-weekly HEMLIBRA prophylaxis demonstrated a statistically significant and clinically meaningful reduction in treated bleeds in an intra-patient comparison of patients receiving HEMLIBRA prophylaxis compared to their prior factor VIII prophylaxis.

While HEMLIBRA very recently became FDA-approved for inhibitor patients, a successful approval process by the agency could lead to an expanded indication for the new therapy.

“HEMLIBRA is the first product to show superior efficacy to factor VIII prophylaxis. These results in people with hemophilia A without inhibitors represent the next step forward in our clinical trial program, which includes the positive HAVEN 1 and interim HAVEN 2 data in people with inhibitors,” said Sandra Horning, MD, chief medical officer and head of Global Product Development at Genentech. “We look forward to working with health authorities to make this treatment available for all people with hemophilia A as soon as possible.”

Source: Genentech press release dated November 19, 2017