Pozelimab — Drug Monograph

Brand names: Veopoz

Drug class: Monoclonal Antibody

Mechanism of Action

Fully human monoclonal antibody that binds complement component C5 and blocks its cleavage into C5a and C5b, preventing assembly of the terminal complement membrane attack complex (C5b-9). This halts terminal-complement–mediated cell injury. In CHAPLE disease (CD55 deficiency with hyperactivation of complement and angiopathic thrombosis and protein-losing enteropathy), pozelimab restores control of the terminal pathway.

FDA Indications

• CHAPLE disease (CD55-deficient protein-losing enteropathy) in adults and pediatric patients 1 year and older (FDA-approved August 2023)

Common Side Effects

• Upper respiratory tract infection
• Headache
• Fatigue
• Injection-site reactions

Clinical Pearl

Pozelimab is a terminal C5 inhibitor FDA-approved for the ultra-rare CHAPLE disease. In paroxysmal nocturnal hemoglobinuria it is investigational, developed as a fixed combination with the C5 siRNA cemdisiran — cemdisiran lowers C5 production so pozelimab can maintain complete blockade at lower, less frequent doses.

Related Therapies & Mechanisms

• Caplacizumab (Cablivi) — Monoclonal Antibody · Supportive Care
• Eculizumab (Soliris) — Monoclonal Antibody · Supportive Care
• Ravulizumab (Ultomiris) — Monoclonal Antibody · Supportive Care
• Alemtuzumab (Campath) — Monoclonal Antibody