On April 1, 2024, AstraZeneca announced that the CFD inhibitor Danicopan received FDA approval for marketing, to be used in combination with C5 antibodies for treating extravascular hemolysis (EVH) in PNH.
Compared with C5 antibody monotherapy, the addition of Danicopan significantly increased hemoglobin levels after 12 weeks of treatment (2.94g/dL vs 0.496g/dL).The study also met multiple secondary endpoints, with 83.3% of patients in the combination therapy group avoiding transfusions after 12 weeks of treatment, compared to only 38.1% in the C5 antibody monotherapy group.
Previously, Apellis' C3 cyclic peptide inhibitor and Novartis' Factor B inhibitor achieved success in succession and outperformed the C5 antibody. The success and approval of CFD+C5 combination therapy will help AstraZeneca further consolidate its position in the complement field, also demonstrating the clinical value of simultaneously blocking downstream C5 and the alternative complement pathway.
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