EMPAVELI (pegcetacoplan) is a complement inhibitor that is primarily indicated for the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare hematologic condition.

(2) Therapeutic Effect of EMPAVELI (pegcetacoplan)

EMPAVELI (pegcetacoplan) is an injectable complement inhibitor that serves as a therapeutic intervention for adults with paroxysmal nocturnal hemoglobinuria (PNH). By targeting the complement cascade, it helps to reduce hemolysis, which is the destruction of red blood cells, a hallmark of PNH. This inhibition of the complement system leads to reduced symptoms, including fatigue, anemia, and the risk of thrombosis, significantly improving patient outcomes and quality of life. It is typically administered as a subcutaneous injection at a dosage of 1,080 mg twice weekly. The exact mechanism of action involves the inhibition of C3, a central component of the complement system, preventing excessive cell destruction. However, EMPAVELI does not directly cure PNH but provides symptomatic relief and disease management.