A recombinant, humanized, bispecific monoclonal antibody

Emicizumab (Hemlibra®), a recombinant, humanized, bispecific monoclonal antibody, restores the function of missing activated factor VIII (FVIII) by bridging FIXa and FX to facilitate effective haemostasis in patients with haemophilia A. Subcutaneous emicizumab is approved in several countries, including in the USA and Japan, for the routine prophylaxis of bleeding episodes in patients with haemophilia A with or without FVIII inhibitors. It is also approved in the EU for the routine prophylaxis of bleeding episodes in patients with haemophilia A with inhibitors or severe haemophilia A without inhibitors. In phase III clinical trials, emicizumab prophylaxis significantly reduced annualized bleeding rates compared with no prophylaxis in adolescents and adults with haemophilia A with or without inhibitors, and prevented or substantially reduced bleeding in children with haemophilia A with or without inhibitors. Emicizumab was also associated with beneficial effects on health-related quality of life and health status, and was generally well tolerated. In view of its convenient route of administration and versatile dosage regimens (maintenance dose of once every 1, 2 or 4 weeks), emicizumab provides an effective and generally well-tolerated alternative to conventional FVIII replacement products for the prophylaxis of bleeding episodes in patients with haemophilia A, regardless of the presence or absence of inhibitors.

is a humanized bispecific antibody for the treatment of haemophilia A, developed by Chugai (a subsidiary of Roche). A Phase I clinical trial found that it was well tolerated by healthy subjects .

In November 2017, it was approved in the United States for treatment of haemophilia A in those who had developed resistance to other treatments. It was subsequently approved by the US FDA in April 2018 under the breakthrough therapy designation for treatment of haemophila A in those who have not developed resistance to other treatments. The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication.

Mechanism of action

Emicizumab binds to both the activated coagulation factor IX and to factor X, mediating the activation of the latter. This is normally the function of coagulation factor VIII, which is missing in haemophilia A patients .

Dosage Forms & Strengths

injection, IV solution (single-dose vials)

30mg/mL

60mg/0.4 mL

105mg/0.7 mL

150mg/mL

Hemophilia A

Indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients aged newborn and older with hemophilia A with or without factor VIII inhibitors

Loading dose: 3 mg/kg SC qWeek for the first 4 weeks

Maintenance dose

1.5 mg/kg SC qWeek, OR

3 mg/kg SC q2Weeks, OR

6 mg/kg SC q4Weeks

Selection of maintenance dose based on healthcare provider preference with consideration of regimens that may increase patient adherence

Discontinue prophylactic use of bypassing agents 1 day before starting prophylaxis

Prophylactic use of factor VIII (FVIII) products may be continued during first week of prophylaxis

Dosage Modifications

Renal impairment

Mild-to-moderate (CrCl 30-89 mL/min): Pharmacokinetics are not affected

Hepatic impairment

Mild-to-moderate (total bilirubin 1X to ≤3X ULN and any AST): Pharmacokinetics are not affected

Dosing Considerations

Self-administration is not recommended for children <7 years .