Complement protein C5 inhibitor Treatment ofcomplement-mediated diseases

Complement-mediated diseases are rare, and potentially life-threatening, in particular the acquired condition of par-oxysmal nocturnal hemoglobinuria. Established treatments include the intravenous complement inhibitors eculizumab and ravulizumab, which targets complement protein C5 and inhibits intravascular hemolysis, and more recently subcuta-neous pegcetacoplan, which targets complement protein C3, inhibiting intravascular and extravascular hemolysis. Treat-ments are however onerous, with physicians and patients wanting improved options. Pozelimab, a subcutaneous weekly self-administered complement inhibitor, binding at C5, has the potential to offer patients effective disease control, while offering independence from homecare or hospital settings. Combination treatment with pozelimab and cemdisiran, a subcutaneous small-interfering RNA (siRNA), which reduces C5 hepatocyte production, has the potential to reduce fre-quency of administration, allowing patients increased auton-omy for disease management.

Automated summary

Complement-mediated diseases, such as paroxysmal nocturnal hemoglobinuria, are rare but life-threatening. Current treatments involve intravenous complement inhibitors, but there is a need for improved options. Pozelimab, a subcutaneous weekly self-administered complement inhibitor, shows potential in offering effective disease control and independence for patients. Combination treatment with pozelimab and cemdisiran may further reduce the need for frequent administration, increasing autonomy for disease management.