Indications
Pegcetacoplan is approved for use in adults with paroxysmal nocturnal hemoglobinuria (PNH). Additionally, it is indicated for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration.
Pharmacodynamics
Pegcetacoplan functions as a complement C3 inhibitor, effectively preventing complement-mediated hemolysis of red blood cells in individuals suffering from paroxysmal nocturnal hemoglobinuria. This medication exhibits a prolonged duration of action, requiring administration twice weekly. It is important for patients to be vaccinated against encapsulated bacteria in accordance with the latest recommendations.
Absorption
The absorption profile of pegcetacoplan is characterized by a median time to maximum concentration (Tmax) ranging from 4.5 to 6.0 days. The pharmacokinetics of pegcetacoplan reach a steady state after approximately 6 to 8 weeks of consistent treatment.
Metabolism
Pegcetacoplan is anticipated to undergo metabolic breakdown into smaller oligopeptides and amino acids. This process is reflective of the typical metabolic pathways for peptide-based therapeutics.
Mechanism of Action
Pegcetacoplan functions by targeting the complement system, a key component implicated in paroxysmal nocturnal hemoglobinuria (PNH), which results from mutations in the PIGA gene. These mutations hinder the production of glycosyl phosphatidylinositol (GPI), essential for anchoring protective proteins like CD55 and CD59 to red blood cells. The absence of these proteins leads to improper regulation of the complement system, causing complement-mediated hemolysis. This pathological process underlies symptoms such as anemia, fatigue, and difficulty breathing in PNH patients.
