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Therapies Targeting the Complement System

المؤلف:  Abbas, A. K., Lichtman, A. H., Pillai, S., & Henrickson, S. E.

المصدر:  Cellular and Molecular Immunology (2026)

الجزء والصفحة:  11E, P316

2026-09-27

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A number of therapeutic agents that target the complement system are currently approved for clinical use.

• Recombinant human C1-INH is used to treat patients with hereditary angioedema.

• Anti-C5 antibodies that block C5 or its cleavage inhibit initiation of the late steps of complement activation. They have been approved for use in patients with paroxysmal nocturnal hemoglobinuria, in patients with the atypical hemolytic uremic syndrome, for the treatment of myasthenia gravis, an autoimmune disease in which autoantibodies recognize the acetylcholine receptor, and also for neuromyelitis optica spectrum disorder, an autoimmune disease linked to autoantibodies to aquaporin.

 • Monoclonal antibodies against human C1s specifically block the classical pathway of complement and have been approved for the treatment of cold agglutinin disease, a disorder in which IgM autoantibodies to a red blood cell antigen cause extravascular hemolysis and anemia.

 • A small molecule competitive inhibitor of the C5a fragment of complement factor 5 binds to C5aR1 receptor and pre vents inflammation driven by C5a. This inhibitor has been approved for the treatment of the autoimmune disease ANCA (antineutrophil cytoplasmic antibody)-associated vasculitis.

• A cyclic tridecapeptide that can bind to C3 and block its activation has been covalently linked to polyethylene glycol to extend its half-life, and this conjugate has been approved for the treatment of paroxysmal nocturnal hemoglobinuria.