Eculizumab-epys (Epysqli)

General information

Class/mechanism: Complement inhibitor; monoclonal antibody that binds to complement protein C5, preventing its cleavage to C5a and C5b and the formation of the terminal complement complex C5b-9. Eculizumab is used in diseases involving abnormal complement-mediated activity, such as paroxysmal nocturnal hemoglobinuria (PNH) and complement-mediated thrombotic microangiopathy (TMA) in patients with atypical hemolytic uremic syndrome (aHUS).

Diseases for which it is established

• Paroxysmal nocturnal hemoglobinuria

Diseases for which it is used

• Atypical hemolytic uremic syndrome
• Cold agglutinin disease

History of changes in EMA indication

• 2023-05-26: Epysqli is indicated in adults and children for the treatment of Paroxysmal nocturnal haemoglobinuria (PNH). Evidence of clinical benefit is demonstrated in patients with haemolysis with clinical symptom(s) indicative of high disease activity, regardless of transfusion history.