Phase III data for Apellis Pharmaceuticals, Inc.’s C3 inhibitor Empaveli (pegcetacoplan) in treatment-naïve paroxysmal nocturnal hemoglobinuria (PNH) appear to offer a largely incremental benefit for the drug given that its broad label already includes such patients. Nevertheless, the data are expected to help with the drug’s uptake, though Alexion Pharmaceuticals Inc.’s C5 inhibitors Soliris (eculizumab) and Ultomiris (ravulizumab-cwvz) are sufficiently entrenched that they could prove difficult to unseat despite Empaveli showing what appears to be better efficacy.
Apellis’ Empaveli Data In Frontline PNH Incremental, But Still Helpful
Data Could Aid Uptake Despite Already Broad Label
While the drug appears more efficacious than Alexion’s C5 inhibitors, those drugs could prove difficult to dethrone given how entrenched they are in PNH therapy.

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