Pozelimab is a medication designed to treat a rare blood disorder called paroxysmal nocturnal hemoglobinuria (PNH). It works by targeting and blocking specific proteins in the immune system that cause the destruction of red blood cells.

Cemdisiran is also a medication used in treating paroxysmal nocturnal hemoglobinuria. It works differently from pozelimab by interfering with the production of certain proteins that contribute to red blood cell destruction.

The combination of pozelimab and cemdisiran is being studied as a potential treatment for patients whose condition is not well controlled with current treatments that target the C5 protein in the blood. This combination therapy aims to provide better control of the breakdown of red blood cells that occurs in patients with this blood disorder.

Paroxysmal Nocturnal Hemoglobinuria (PNH) – A rare blood disorder where the body’s immune system attacks and destroys red blood cells prematurely. In this condition, red blood cells break down faster than the body can replace them, a process called hemolysis. The condition is caused by a genetic mutation that occurs in blood-forming stem cells. PNH can develop at any age but is most often diagnosed in young adults. The name comes from the fact that symptoms were historically noted to be worse at night, though we now know the destruction of red blood cells occurs throughout the day. Patients may experience episodes where symptoms become more severe, followed by periods of relative stability.