C5inhibitorokkal

C5 inhibitorok are a class of drugs that target the fifth component of the complement system, known as C5. The complement system is a part of the innate immune system that plays a crucial role in defending the body against pathogens and clearing damaged cells. It is a complex cascade of proteins that can be activated through various pathways. When activated, these proteins can lead to inflammation, cell lysis, and opsonization.

The C5 protein is a critical juncture in the complement cascade. Cleavage of C5 by enzymes called C5 convertases leads to the formation of C5a and C5b. C5a is a potent anaphylatoxin, contributing to inflammation and attracting immune cells. C5b initiates the formation of the membrane attack complex (MAC), which can directly lyse target cells. By inhibiting C5, these drugs block the downstream consequences of C5 cleavage, thereby dampening the inflammatory response and preventing cell lysis mediated by the complement system.

C5 inhibitorok are primarily used in the treatment of certain rare, chronic, and debilitating diseases where the complement system is dysregulated and contributes to pathology. These include conditions such as paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and generalized myasthenia gravis (gMG). By selectively blocking the C5 protein, these therapies aim to reduce disease activity, prevent organ damage, and improve patient quality of life. Their development represents a significant advancement in targeted immunomodulatory therapies.