The US Food and Drug Administration has approved danicopan (Voydeya, AstraZeneca) as an add-on therapy to treat extravascular hemolysis in adults receiving ravulizumab orPNH is a rare blood disorder affecting 1-10 individuals per million. The condition, which eliminates red blood cells and leads to blood clots and impaired bone marrow function, can cause life-threateningRavulizumab and eculizumab, also both made by AstraZeneca, inhibit the destruction of red blood cells. However, 10%-20% of patients treated with the antibody infusions experience significant extravascular hemolysis, in which these surviving red blood cells are eliminated by the spleen and liver. Extravascular hemolysis can lead to ongoing anemia, which can lead patients to require blood transfusions. Danicopan, an investigational, first-in-class, oral complement factor D inhibitor, is designed to control intravascular hemolysis and prevent extravascular hemolysis
The US Food and Drug Administration has approved danicopan (Voydeya, AstraZeneca) as an add-on therapy to treat extravascular hemolysis in adults receiving ravulizumab or PNH is a rare blood disorder affecting 1-10 individuals per million. The condition, which eliminates red blood cells and leads to blood clots and impaired bone marrow function, can cause life-threateningRavulizumab and eculizumab, also both made by AstraZeneca, inhibit the destruction of red blood cells .
However, 10%-20% of patients treated with the antibody infusions experience significant extravascular hemolysis, in which these surviving red blood cells are eliminated by the spleen and liver. Extravascular hemolysis can lead to ongoing anemia, which can lead patients to require blood transfusions. Danicopan, an investigational, first-in-class, oral complement factor D inhibitor, is designed to control intravascular hemolysis and prevent extravascular hemolysi
FDA Danicopan Add-On Therapy Hemolysis Treatment Blood Disorder Astrazeneca Ravulizumab PNH Red Blood Cells Anemia
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