Expert: Humanized monoclonal antibody that binds complement C5, preventing cleavage to C5a and C5b and inhibiting terminal complement-mediated hemolysis and inflammation.
Everyday: Blocks a protein in the immune system that mistakenly attacks the body's own red blood cells.
Targets: ["C5"]
Approved for CHAPLE disease (CD55-deficient protein-losing enteropathy). Ultra-rare indication. C5 inhibitor (same target as Soliris/Ultomiris from AZN). CHAPLE patients lose complement regulator CD55, causing uncontrolled complement activation → intestinal damage. Revenue not separately reported.
{"keyRisks":[{"category":"Meningococcal infections","description":"C5 complement inhibition increases susceptibility to Neisseria meningitidis. Vaccinate before starting. REMS program required.","incidenceRate":"Class risk"},{"category":"Other serious infections","description":"Complement inhibition impairs defense against encapsulated bacteria (pneumococcus, H. influenzae).","incidenceRate":"Theoretical risk"},{"category":"Upper respiratory tract infection","description":"Common AE in clinical trials.","incidenceRate":"21%"},{"category":"Headache","description":"Reported in trials.","incidenceRate":"14%"}],"monitoring":["Meningococcal vaccination status","Signs of meningococcal infection (fever, headache, stiff neck, rash)","Vaccinate against pneumococcus, H. influenzae"],"classWarnings":["C5 complement inhibitor: meningococcal infection risk (same class as eculizumab/Soliris)","REMS program: meningococcal vaccination required"],"hasBoxedWarning":true,"boxedWarningText":"SERIOUS MENINGOCOCCAL INFECTIONS: Life-threatening or fatal meningococcal infections have occurred. Vaccinate patients with meningococcal vaccines at least 2 weeks prior to first dose. Monitor for early signs of meningococcal infections."}