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How does Empaveli work?

Medically reviewed by Carmen Fookes, BPharm. Last updated on Sep 8, 2021.

Official answer

by Drugs.com

Empaveli has effects on 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. Empaveli binds to complement protein C3 and prevents C3 from splitting into C3a and C3b. This helps to stop intravascular hemolysis (IVH or the destruction of red blood cells in the circulation) and extravascular hemolysis (EVH or the breakdown of red blood cells in the spleen, bone marrow, or liver by macrophages). Empaveli is the only treatment that targets C3 and may prevent both forms of red blood cell destruction (IVH and EVH).

Empaveli is a prescription medicine used to treat adults with a condition called 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.


References
  • Empaveli (pegcetacoplan) Updated 07/2021. Apellis Pharmaceuticals, Inc. https://www.drugs.com/pro/empaveli.html
  • Paroxysmal Nocturnal Hemoglobinuria (PNH) The Sidney Kimmel Comprehensive Cancer Center. John Hopkins Medicine. https://www.hopkinsmedicine.org/kimmel_cancer_center/types_cancer/paroxysmal_nocturnal_hemoglobinuria_PNH.html#:~:text=Paroxysmal%20nocturnal%20hemoglobinuria%20(PNH)%20is,of%20the%20three%20blood%20components).

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