Crovalimab: a new era in paroxysmal nocturnal hemoglobinuria management

Abstract

Crovalimab, a new promising drug for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) has emerged. This marks a significant advancement in the treatment of PNH and potentially other complement-mediated disorders. PNH is characterized by complement-mediated hemolysis, thrombosis, and bone marrow failure, leading to severe morbidity and mortality in affected individuals. PNH arises from a somatic mutation in the PIGA gene, leading to the loss of glycosylphosphatidylinositol (GPI)-anchored proteins on blood cells, making them vulnerable to complement-mediated destruction. Crovalimab is a new anti-C5 recycling antibody that offers a promising treatment by being administered subcutaneously every 4 weeks at a low volume. Clinical trials such as COMMODORE 3 have shown crovalimab's effectiveness and high tolerability in PNH patients who have not previously used a C5 inhibitor. Crovalimab has been proven effective in maintaining hemoglobin levels, reducing the need for transfusions, and improving patient outcomes by inhibiting terminal complement activation.

Keywords: PIGA gene; PNH; anti-c5 antibody; commodore trials; complement-mediated destruction; crovalimab; glycosylphosphatidylinositol; hemolysis; paroxysmal nocturnal hemoglobinuria; terminal complement activation; transfusion reduction.