FDA approves Empaveli to treat paroxysmal nocturnal hemoglobinuria.-Apellis Pharmaceuticals

Apellis Pharmaceuticals, Inc. announced that the FDA has approved Empaveli (pegcetacoplan), the first and only targeted C3 therapy for treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH). Empaveli is approved for use in adults with PNH who are treatment naïve as well as patients switching from the C5 inhibitors Soliris (eculizumab) and Ultomiris (ravulizumab). The approval of Empaveli is based on results from the head-to-head Phase III PEGASUS study, which were recently published in the New England Journal of Medicine. In the PEGASUS study, Empaveli met the primary endpoint, demonstrating superiority to Soliris for the change from baseline in hemoglobin level at Week 16 with an adjusted mean increase of 3.84 g/dL of hemoglobin (p<0.0001). additionally, empaveli met non-inferiority compared to soliris on the endpoint of transfusion avoidance. eighty five percent of empaveli-treated patients were transfusion free over 16 weeks versus 15% of soliris-treated patients.. the prescribing information for empaveli contains a boxed warning. empaveli may increase the risk of meningococcal and other serious infections caused by encapsulated bacteria that may become rapidly life threatening or fatal if not recognized and treated early. a risk evaluation and mitigation strategy (rems) has been approved by the fda for empaveli. prescribers must counsel patients about the risk of serious infection, provide patients with the rems educational materials, and ensure patients are vaccinated against encapsulated bacteria.>