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Zilucoplan is a 15-amino acid, synthetic macrocyclic peptide that acts as a complement inhibitor. It is approved for the treatment of generalized myasthenia gravis (gMG) in adults who are anti-acetylcholine receptor (AChR) antibody positive. Zilucoplan works by binding to the complement protein C5 (C5) with high affinity, inhibiting its cleavage into C5a and C5b, and preventing the formation of the terminal complement complex (C5b-9). This mechanism reduces complement-mediated damage at the neuromuscular junction, which is implicated in gMG pathophysiology. The drug can be self-administered once daily via subcutaneous injection and has demonstrated rapid and sustained inhibition of complement activity in clinical trials[1][2][3][4][5][6][7].
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