FDA Approves New Once-Weekly Prophylactic Treatment For Hemophilia A With Inhibitors

November 16th, 2017

On November 16, the FDA approved HEMLIBRA® (emicizumab-kxwh) for routine prophylaxis (preventative treatment) to prevent or reduce the frequency of bleeding episodes in adults and children with hemophilia A with factor VIII inhibitors.

News in brief

  • HEMLIBRA is the first new FDA-approved medicine in nearly 20 years to treat hemophilia A with inhibitors
  • Only treatment option that can be self-administered once-weekly by injection under the skin (subcutaneously)
  • 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

Sandra Horning, M.D.

"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 almost 20 years. 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.”

“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. 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.”