The U.S. Food and Drug Administration (FDA) has approved the targeted C3 inhibitor pegcetacoplan for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH).
This approval is based on results from a study of 80 patients with PNH and severe anemia who were being treated with eculizumab. During the first four weeks of the study, patients received pegcetacoplan 1,080 mg twice weekly in addition to eculizumab. After the initial four-week period, patients were randomized to receive either pegcetacoplan or eculizumab for 16 weeks. At baseline, the average hemoglobin level across both treatment groups was 8.7 g/dL. During the 16 weeks of treatment, patients in the pegcetacoplan arm had an average increase in their hemoglobin of 2.4 g/dL, compared with an average decrease of 1.5 g/dL for patients in the eculizumab group.
Patients receiving pegcetacoplan may develop meningococcal infections and be predisposed to serious infections, especially those caused by encapsulated bacteria. Common adverse events associated with pegcetacoplan include injection site reactions, infections, diarrhea, abdominal pain, respiratory tract infection, viral infection, and fatigue.
Pegcetacoplan was granted priority review, fast track, and orphan drug designations for this indication. The treatment is only available through a restricted program under a risk evaluation and mitigation strategy.