FDA Approves Apellis’ Empaveli for Rare Blood Disease

Apellis Pharmaceuticals’ Empaveli (pegcetacoplan) has received FDA approval for treating patients with paroxysmal nocturnal hemoglobinuria, a rare, genetic blood disease characterized by the destruction of red blood cells.

A targeted C3 immune protein therapy, Empaveli is designed to boost hemoglobin and reduce blood transfusion requirements. The approval was supported by positive results from a phase 3 trial showing Empaveli boosted hemoglobin levels from baseline at week 16.

Empaveli is approved for adults who are treatment-naïve and for those changing regimens from Alexion Pharmaceuticals’ Soliris (eculizumab) Soliris and Alexion’s Ultomiris (ravulizumab).