Phase III APPLY-PNH trial met its two primary endpoints for superiority versus anti-C5 treatment in adult paroxysmal nocturnal hemoglobinuria (PNH) patients with residual anemia despite prior anti-C5 treatment1
Iptacopan is an investigational, first-in-class alternative complement pathway inhibitor that specifically inhibits factor B, with potential to become the first oral monotherapy in PNH1-5
PNH has a significant unmet need not addressed by anti-C5 therapies; despite treatment with anti-C5s, a large proportion of people with PNH remain anemic, fatigued and dependent on blood transfusions
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