Home » FDA Approves Apellis’ Empaveli for Rare Blood Disease
FDA Approves Apellis’ Empaveli for Rare Blood Disease
Drugs Submissions and Approvals
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).