FDA New Drug and Biologic Approvals -- 2017 Year-in-Review

Mary L Windle, PharmD


January 11, 2018

In This Article

Hemlibra (emicizumab-kxwh)


Routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients with hemophilia A with factor VIII inhibitors.


Bispecific factor IXa- and factor X-directed antibody that bridges activated FIX and FX in order to restore the function of missing activated FVIII necessary for effective hemostasis.


3 mg/kg SC every week for the first 4 weeks, then 1.5 mg/kg SC every week thereafter.


Approval was based on the HAVEN 1 and 2 clinical trials. The HAVEN 1 trial included 109 adult and adolescent males aged 12 years or older who had hemophilia A with inhibitors. Patients taking emicizumab experienced about 2.9 treated bleeding episodes per year, compared with about 23.3 treated bleeding episodes per year for patients who did not receive prophylactic treatment, representing an 87% reduction in the rate of treated bleeding episodes (P<0.001). Emicizumab-treated patients also reported an improvement in hemophilia-related symptoms (painful swellings and joint pain) and physical functioning (pain with movement and difficulty walking).

Interim results from the single-arm HAVEN 2 study in children aged younger than 12 years with hemophilia A with inhibitors who received emicizumab prophylaxis are consistent with the positive results from the HAVEN 1 study. After a median observation time of 12 weeks, only 1 of the 19 study patients receiving emicizumab reported a treated bleed.


Oldenburg J, et al. Emicizumab prophylaxis in hemophilia A with inhibitors. N Engl J Med. 2017 Aug 31. 377 (9):809-18.

HAVEN 2 interim data. Presented at the 26th Congress of the International Society on Thrombosis and Haemostasis (ISTH) Meeting. Berlin, Germany. July 8-13, 2017.


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