The US FDA has approved danicopan as an add-on therapy to C5 inhibitors ravulizumab or eculizumab for treating extravascular hemolysis (EVH) in adult patients with paroxysmal nocturnal hemoglobinuria (PNH). Danicopan is a complement Factor D inhibitor developed to address the problem of PNH patients experiencing EVH while on C5 inhibitor treatment. Danicopan also prevents fragmentation of complement Factor B and thus C3 fragment-mediated EVH. Clinical evidence In phase 3 clinical trial conducted on 63 PNH patients, danicopan plus ravulizumab or eculizumab improved hemoglobin level (2.9 g/dL vs.
0.5 g/dL), transfusion avoidance (83.3% vs. 38.1%), and Functional Assessment of Chronic Illness Therapy – Fatigue score (8.0 vs. 1.9) in patients compared to placebo plus ravulizumab or eculizumab at week 12. Dosage and administration 150 mg; three times a day orally with or without food It can…
