Emicizumab (Hemlibra)

Mechanism of action

A recombinant, humanized, bispecific monoclonal antibody that bridges activated factor IX and factor X to restore the function of missing activated factor VIII.

Diseases for which it is used

• Acquired factor VIII inhibitor in the setting of inherited factor VIII deficiency

History of changes in FDA indication

• 2017-11-16: Approved "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."
• 2018-10-04: Approval expanded "for prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients (ages newborn and older) with hemophilia A (congenital factor VIII deficiency) with or without factor VIII (FVIII) inhibitors. (requirement for inhibitor to be present was removed)

History of changes in EMA indication

• 2018-02-23: Initial authorisation
• Uncertain date: Hemlibra is indicated for routine prophylaxis of bleeding episodes in patients with haemophilia A (congenital factor VIII deficiency) with factor VIII inhibitors. Hemlibra can be used in all age groups.
• 2023-03-06: Indicated revised to include patients with haemophilia A (congenital factor VIII deficiency) without factor VIII inhibitors who have severe disease (FVIII < 1%) or moderate disease (FVIII at least 1% and up to 5%) with severe bleeding phenotype. Hemlibra can be used in all age groups.

Also known as

• Code name: ACE910
• Generic name: emicizumab-kxwh
• Brand name: Hemlibra