Pegcetacoplan, the first C3 inhibitor for the treatment of PNH, was recently approved by the FDA and EMA.21, –23 New treatment strategies will aim to address some of the challenges of current therapies (e.g. EVH-mediated anemia, patient convenience, and HRQoL).98 Future PNH therapy should seek to maintain terminal complement suppression and improve HRQoL in this chronic and potentially devastating disease. The gene discussed is C3; the disease is anemia (phenotype).