Apellis says EMPAVELI provided long-term control of PNH

Apellis Pharmaceuticals and Sobi presented post hoc data that reinforce the long-term efficacy and safety of EMPAVELI in adults with paroxysmal nocturnal hemoglobinuria for up to three years. The data were reported during an oral presentation at the American Society of Hematology Annual Meeting. The analysis integrated data across the Phase 3 PEGASUS and PRINCE studies and the long-term extension study. After starting treatment with EMPAVELI, key markers of disease rapidly improved and were sustained in both treatment-naive patients and patients previously treated with eculizumab. Improvements in hemoglobin reached normal or near-normal levels, and mean lactate dehydrogenase was maintained below the upper limit of normal. Additionally, 67% of treatment-naive patients from PRINCE were transfusion free for up to 2.5 years, and 52% of patients from PEGASUS remained transfusion free for up to 3 years. Less than 25% of patients were transfusion free in the year prior to entering the PRINCE and PEGASUS studies. The safety profile was consistent with previous clinical study results, and no new or unexpected safety findings were identified. Approximately 4.5% of patients experienced a serious adverse event deemed related to treatment with pegcetacoplan. No meningococcal infections were reported. The prescribing information for EMPAVELI contains a boxed warning, which states that 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.