The approval is based on the head-to-head PEGASUS study, which showed that complement C3 inhibitor Empaveli (pegcetacoplan) was better than Soliris (eculizumab) at improving haemoglobin levels in adults with PNH, an autoimmune disease characterised by the destruction of red blood cells and anaemia.
This study enrolled 80 adult PNH patients who had been on intravenous Soliris and were stable for at least three months with a haemoglobin level of less than 10.5 g/dL at baseline.
Patients received twice-weekly subcutaneous doses of Empaveli in addition to Soliris over four weeks and were then randomised to receive Apellis’ drug or their current Soliris dose for the main treatment phase of the trial.
