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HEMLIBRA® Receives Breakthrough Designation from FDA

April 17, 2018

Genentech has announced that the US Food and Drug Administration (FDA) has granted “breakthrough” therapy designation to HEMLIBRA® for people with hemophilia A without inhibitors. Breakthrough drugs are put on a fast-track approval program and given intensive guidance from the FDA. The therapy was co-developed by Genentech, Chugai and Roche.

HEMLIBRA is already FDA-approved 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 2017. While existing factor therapies are administered intravenously, HEMLIBRA is novel in that it is delivered subcutaneously through an injection just under the skin.

The designation was based on data from the phase III, HAVEN 3 clinical study in patients 12 years or older with hemophilia A without inhibitors. 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 who were previously treated with factor VIII therapy either on-demand or on prophylaxis.

According to a Genentech press release posted on April 16, 2018, HEMLIBRA prophylaxis administered subcutaneously every week or every two weeks showed a “statistically significant and clinically meaningful” reduction in treated bleeds compared to no prophylaxis. In addition, an intra-patient comparison (same patient at a different time) showed that once-weekly HEMLIBRA prophylaxis was superior to prior factor VIII prophylaxis. This led to a “statistically significant and clinically meaningful” reduction in treated bleeds. The most common adverse events reported were injection site reactions. No thrombotic microangiopathy or thrombotic events were observed during the study.

“HEMLIBRA is the first medicine to show superior efficacy compared to factor VIII prophylaxis, the standard of care for people with hemophilia A without inhibitors, in an intra-patient comparison,” said Sandra Horning, MD, chief medical officer and head of Global Product Development. “We look forward to working with health authorities to make HEMLIBRA available to people without inhibitors as soon as possible, and we are excited to share this news with the community as we join in celebrating World Hemophilia Day.”

Source: Genentech press release dated April 16, 2018