FDA Approves New Option For Some Patients With Hemophilia A

The US Food and Drug Administration recently approved Hemlibra (emicizumab-kxwh) to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A who developed Factor VIII (FVIII) inhibitors.

Hemlibra is administered once a week as a subcutaneous injection and works by bridging other Factors in blood to restore blood clotting.
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Approval of Hemlibra was determined using data from 2 clinical trials. In the first trial, patients who received Hemlibra experienced an 87% reduction in the rate of treated bleeds and improvements in symptoms and functioning. Similarly, 87% of patients who received Hemlibra in the second trial did not experienced a bleeding episode that required treatment.

Common adverse effects included injection site reactions, headache, and arthralgia.

In addition, the drug has a boxed warning on severe blood clots, which has been observed in patients who were administered rescue treatment to treat bleeds for 24 hours or more while receiving Hemlibra.

—Melissa Weiss

Reference:

FDA approves new treatment to prevent bleeding in certain patients with hemophilia A [press release]. 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 16, 2017.