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.

Render illustration of Clinical Trial title on medical documents
Results of Apellis' PRINCE study of Empaveli seen as having incremental benefit for already-approved drug • Source: Shutterstock

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.

The Waltham, MA-based Apellis and Stockholm-based Swedish Orphan Biovitrum AB (Sobi) announced on 25 May topline results from the...

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