The acquisition adds two clinical-stage oral small molecule Factor D inhibitors to Alexion’s pipeline and provides the foundation and expertise for a broader oral Factor D inhibition development platform with the potential to treat numerous additional complement-mediated diseases including PNH.
Alexion will continue development of Achillion’s oral Factor D inhibitor portfolio, which includes two clinical-stage medicines-in-development – danicopan (ACH-4471) and ACH-5228 – as well as multiple compounds in preclinical development. Phase 3 development is being initiated for danicopan as an add-on therapy for PNH patients with extravascular hemolysis (EVH). Danicopan is also in Phase 2 development for C3G, and ACH-5228 is in Phase 2 development for patients with PNH.
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