Chapter 14: Soliris (Eculizumab): Discovery and Development Check Access

Eculizumab, marketed as ‘Soliris’ by Alexion Pharmaceuticals Inc., is a humanised, Fc-engineered (low effector function), IgG2/4 hybrid monoclonal antibody that blocks the activity of human complement factor C5. This activity enables eculizumab to potently suppress the pro-inflammatory and cytolytic effects of the terminal complement system, which are key factors in the pathology of the rare haemolytic disease paroxysmal nocturnal haemoglobinuria (PNH). This antibody therapeutic has become an important example of a potential therapeutic molecule that was originally developed for a series of common inflammatory diseases (rheumatoid arthritis, lupus, etc.), but was repurposed with great success to become a first-in-class complement inhibitor in the rare disease space.