Pegcetacoplan-Empaveli’s listing status and market feedback

Pegcetacoplan is a therapeutic drug targeting complement C3, developed by Apellis Pharmaceuticals. After years of clinical research and trials, it was approved by the U.S. Food and Drug Administration (FDA) on May 14, 2021, for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in adults. This approval marks Empaveli as the first complement C3-targeted therapy, providing a new treatment option for adult patients with PNH.

PNH is a rare, chronic and life-threatening blood disease, which mainly manifests as intravascular and extravascular hemolysis, leading to the destruction of red blood cells, which in turn causes anemia, fatigue, hemoglobinuria and other symptoms. People with PNH often experience increasing health problems over time, and many patients require frequent blood transfusions to maintain normal hemoglobin levels. Traditionally, treatments for PNH have focused on slowing the hemolysis process through C5 inhibitors. However, Empaveli, as an innovative drug targeting complement C3, has shown a more significant therapeutic effect by inhibiting the excessive activation of the complement system at an earlier stage.

Empaveli's efficacy has been well documented in clinical trials. In the head-to-head Phase 3 PEGASUS study, Empaveli demonstrated significant advantages in increasing hemoglobin levels and reducing the need for blood transfusions in patients with PNH. Compared with the traditional C5 inhibitor Soliris, Empaveli can improve anemia symptoms in PNH patients more quickly and effectively, and within 16 weeks, 85% of patients treated with Empaveli did not need blood transfusions, while only 15% of patients in the eculizumab treatment group achieved this goal within the same time. This result provides a solid foundation for the application of Empaveli in the treatment of PNH.

Reference materials:https://empaveli.com