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FDA Approves Hemlibra to Prevent Bleeding
FDA news release; 2017 Nov 9
The FDA has approved Hemlibra (emicizumab-kxwh) to prevent or reduce the frequency of bleeding episodes in certain patients with hemophilia A.
Indications: Hemlibra is a bispecific factor IXa- and factor X-directed antibody indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A (congenital factor VIII deficiency) with factor VIII inhibitors.
Dosage and administration: Recommended dose is 3 mg/kg by subcutaneous injection once weekly for the first 4 weeks, followed by 1.5 mg/kg once weekly.
Efficacy and safety: Approval is based on results from 2 clinical trials. The first showed that Hemlibra-treated patients experienced ~3 treated bleeding episodes/year, vs ~23/year in those who were not treated. The second showed that 87% of patients taking Hemlibra did not experience a bleeding episode that required treatment.
Side effects/risks: The most common adverse reactions are injection site reactions, headache, and arthralgia.
FDA approves new treatment to prevent bleeding in certain patients with hemophilia A. [news release]. Silver Spring, MD: FDA. November 16, 2017. https://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm585567.htm?utm_campaign=11162017_Hemophilia%20A%20treatment&utm_medium=email&utm_source=Eloqua. Accessed November 20, 2017.
Hemlibra [package insert]. South San Francisco, CA: Genentech, Inc. 2017. https://www.accessdata.fda.gov/drugsatfda_docs/label/2017/761083s000lbl.pdf. Accessed November 20, 2017.
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