Emicizumab-kxwh (Hemlibra) for the prevention or reduction of bleeding episodes for adult and pediatric patients with hemophilia A with Factor VIII inhibitors.
The approval, is significant since Factor VIII inhibitors can make available treatments less effective.
Emicizumab-kxwh bridges other Factors in the blood to restore clotting function and is injected subcutaneously as a weekly prophylactic treatment.
Safe and effective agent, with the most common adverse events were injection site reactions, headache, and arthralgia.
In patients with hemophilia A who do not have factor VIII inhibitors, emicizumab therapy outperformed factor VIII prophylaxis, according to a results of a randomized, open-label, phase 3 trial.
Emicizumab, a humanized bispecific antibody that mimics FVIII activity by binding to both activated coagulation FIX and factor X (FX), mediating the activation of FX and facilitating blood clot formation.
Emicizumab, given subcutaneously, is approved to treat patients with hemophilia A who had developed inhibitors to FVIII and could no longer receive FVIII as a therapy.
Such patients account for between 25 and 40% of patients with severe hemophilia A.
It indication has been expanded to the treatment of all patients with severe hemophilia A – either with and without inhibitors – including newborns.
Emicizumab makes a patient with severe hemophilia more like a patient with mild disease.
Patients treated with emicizumab had a significantly lower bleeding rate than without prophylaxis, and more than half of the treated patients had no treated bleeding events during the trial.
It is a monoclonal antibody that joins activated factor IX with factor X.
This combination restores hemostasis by replacing missing factor VII
Approved for patients with hemophilia A and anti-factor VIII alloantibodies (inhibitors).
The most common adverse event associated is injection-site reactions.
No thrombotic events, thrombotic microangiopathies, or deaths occurred with use.
Patients taking emicizumab-kxwh have an 87% reduction in the rate of treated bleeding episodes per year, compared with patients not receiving prophylactic treatment.(Haven trials).
On a trial in children 87% of the children receiving emicizumab-kxwh did not experience a bleeding episode that required treatment.
Possibility of thrombotic microangiopathy and thromboembolism in patients given an activated prothrombin complex concentrate rescue treatment for 24 hours or more while taking
Administered once weekly, although 4 week adminstration protocols may be as effective.