Empaveli (pegcetacoplan) is an immunosuppressant that is given by subcutaneous (under the skin) infusion, which may be used to treat adults with paroxysmal nocturnal hemoglobinuria (PNH). PNH is a rare acquired, life-threatening disease of the blood that is characterized by the destruction of red blood cells (hemolytic anemia), blood clots (thrombosis), and impaired bone marrow function.
Empaveli for PNH works by binding to specific proteins in the complement system, which is a part of the immune system that enhances (or complements) the ability of antibodies and phagocytic cells to clear microbes and damaged cells from an organism, promote inflammation, and attack the pathogen's cell membrane.
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