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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The first targeted C3 therapy was approved by the US FDA for treatment-naïve patients as well as those switching from Alexion's Soliris or Ultomiris.
Sobi is paying big bucks to tap into the potential of Apellis's pegcetacoplan, a targeted C3 therapy for the treatment of multiple rare diseases with high unmet need which the partners say impact more than 275,000 patients globally.
Phase II results look good for one of the first-in-class product’s potential indications, C3 glomerulopathy. The product was highlighted late last year as an up-and-coming pipeline asset.