FDA Approves Genentech’s HEMLIBRA (emicizumab-kxwh) for Hemophilia A with Inhibitors

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today that the U.S. Food and Drug Administration (FDA) has approved HEMLIBRA® (emicizumab-kxwh) for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with factor VIII inhibitors. Nearly one in three people with severe hemophilia A can develop inhibitors to factor VIII replacement therapies, putting them at greater risk for life-threatening bleeds or repeated bleeds that can cause long-term joint damage. In two of the largest pivotal clinical studies for people with hemophilia A with inhibitors, HEMLIBRA was shown to substantially reduce bleeds in adults and children.

“People with hemophilia A who develop inhibitors face significant challenges preventing bleeds and typically require infusions of medicine multiple times a week, which can be especially difficult for young children and their families,” said Guy Young, M.D., director of Hemostasis and Thrombosis Program, Children’s Hospital Los Angeles, and professor of Pediatrics, University of Southern California Keck School of Medicine, Los Angeles, California. “This new medicine has been shown to reduce the frequency of bleeds compared to the currently available medicines and only needs to be injected once a week. This could make a meaningful difference for these children.”

“Before HEMLIBRA, my 7-year-old son needed intravenous infusions that could take up to two hours at least three times a week, so our lives revolved around his treatment,” said Amber Hill, mother of a young boy with hemophilia A with inhibitors. “With HEMLIBRA, he now has an injection once a week that he has proudly learned to administer himself to help prevent bleeds. Not only has he had fewer bleeds compared to his prior treatment, he has more time to be a kid and we have more quality time as a family because of the new treatment schedule.”

In the Phase III HAVEN 1 study, people 12 years of age or older with hemophilia A with inhibitors who received HEMLIBRA prophylaxis had a statistically significant reduction in treated bleeds of 87 percent (95 percent CI: 72.3; 94.3, p<0.0001) compared to those who received no prophylaxis. In a first-of-its-kind intra-patient analysis, HEMLIBRA prophylaxis resulted in a statistically significant reduction in treated bleeds of 79 percent (95 percent CI: 51.4; 91.1, p=0.0003) compared to previous treatment with bypassing agent (BPA) prophylaxis collected in a non-interventional study (NIS) prior to enrollment.

Interim results from the pivotal HAVEN 2 study in children younger than 12 years of age with hemophilia A with inhibitors showed that 87 percent (95 percent CI: 66.4; 97.2) of children who received HEMLIBRA prophylaxis experienced zero treated bleeds. In an intra-patient analysis of 13 children who had participated in the NIS, HEMLIBRA prophylaxis resulted in a 99 percent reduction in treated bleeds compared to previous treatment with a BPA either as prophylaxis (n=12) or on-demand (n=1). The most common adverse events (AEs) occurring in 10 percent or more of people treated with HEMLIBRA in pooled studies were injection site reactions, headache and joint pain (arthralgia).

“Today’s approval of HEMLIBRA represents an important advancement for people with hemophilia A with inhibitors, who have struggled to manage their bleeding disorder and haven’t had a new medicine in nearly 20 years,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “We believe HEMLIBRA will improve protection against bleeds and reduce the treatment administration burden for people with hemophilia A with inhibitors, and we are committed to helping them access this medicine.”

HEMLIBRA will be available to people in the U.S. shortly after approval. Genentech is committed to helping people with hemophilia A with inhibitors access HEMLIBRA and will be offering comprehensive services to help minimize barriers to access and reimbursement. Patients can call (866) HEMLIBRA (436-5427) for more information. For people who qualify, Genentech also plans to offer patient assistance programs through Genentech Access Solutions.

More information is available at (866) 4ACCESS/(866) 422-2377 or http://www.Genentech-Access.com.

November 16, 2017


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