A review article reports that an increasing number of agents that affect the complement system are in development, including some for common diseases. While many agents target rare diseases, “the substantial pipeline of up-and-coming treatment options” will provide new opportunities.
“The complement system is recognised as a protector against blood-borne pathogens and a controller of immune system and tissue homoeostasis,” the authors write. “However, dysregulated complement activity is associated with unwanted or non-resolving immune responses and inflammation, which induce or exacerbate the pathogenesis of a broad range of inflammatory and autoimmune diseases. Although the merit of targeting complement clinically has long been acknowledged, the overall complement drug approval rate has been modest. However, the success of the humanised anti-C5 antibody eculizumab in effectively treating paroxysmal nocturnal haemoglobinuria and atypical haemolytic syndrome has revitalised efforts to target complement therapeutically. Increased understanding of complement biology has led to the identification of novel targets for drug development that, in combination with advances in drug discovery and development technologies, has resulted in a surge of interest in bringing new complement therapeutics into clinical use.”