Pozelimab plus cemdisiran achieved intravascular hemolysis control in patients with paroxysmal nocturnal hemoglobinuria. The FDA has accepted the BLA for crovalimab, an investigational treatment for paroxysmal nocturnal hemoglobinuria. Iptacopan, a complement factor B inhibitor, was effective in patients with PNH with active hemolysis despite eculizumab use. Take a look at these important studies relevant to anemia, venous thromboembolism, sickle cell disease, and PNH. A study assessed survival and adverse events in patients with PNH treated with eculizumab. Pegcetacoplan is a cost-effective alternative for the treatment of paroxysmal nocturnal hemoglobinuria. The U.S. FDA has accepted a Biologics License Application for a novel drug to treat paroxysmal nocturnal hemoglobinuria.