Eculizumab

Eculizumab

Eculizumab is a monoclonal antibody that is used in the treatment of various autoimmune diseases. It is marketed under the trade name Soliris by Alexion Pharmaceuticals.

Mechanism of Action

Eculizumab works by inhibiting the complement system, a part of the immune system that enhances the ability of antibodies and phagocytic cells to clear microbes and damaged cells from an organism. It does this by binding to the complement protein C5, preventing it from splitting into C5a and C5b. This inhibits the inflammatory response and the formation of the membrane attack complex, which is responsible for cell lysis.

Uses

Eculizumab is used in the treatment of paroxysmal nocturnal hemoglobinuria (PNH), a rare, life-threatening disease of the blood characterized by hemolysis, thrombosis, and peripheral cytopenia. It is also used in the treatment of atypical hemolytic uremic syndrome (aHUS), another rare disease that causes abnormal blood clots to form in small blood vessels in the kidneys. More recently, it has been approved for use in treating neuromyelitis optica spectrum disorder (NMOSD), a rare neurological condition.

Side Effects

Common side effects of eculizumab include headache, nausea, anemia, and fatigue. Serious side effects can include life-threatening infections, such as meningococcal infections, due to the drug's effect on the immune system.

History

Eculizumab was first approved by the Food and Drug Administration (FDA) in 2007 for the treatment of PNH. It was the first drug approved for this condition. In 2011, it was approved for the treatment of aHUS. In 2019, it was approved for the treatment of NMOSD.

See Also

• Monoclonal antibodies
• Autoimmune diseases
• Paroxysmal nocturnal hemoglobinuria
• Atypical hemolytic uremic syndrome
• Neuromyelitis optica spectrum disorder