The data show that EMPAVELI, the first and only targeted C3 therapy, demonstrated robust and sustained improvements in key markers of disease across a broad population of PNH patients for up to two years.
'These results highlight the meaningful, long-term improvements that treatment with EMPAVELI can make in the lives of PNH patients, regardless of their severity of disease or prior treatment,' said Peter Hillmen, M.B. Ch.B., Ph.D., head of hematology engagement at Apellis. 'EMPAVELI continues to demonstrate its potential to become a new standard of care for adults living with PNH, including the ability to achieve normalization across key markers of disease.'
The new results reinforce the positive efficacy and safety of EMPAVELI in both treatment-naive patients and patients who had previously been treated with eculizumab, a C5 inhibitor, across all five prior PNH clinical studies. After being treated with EMPAVELI for up to two years, mean hemoglobin was sustained at near-normal levels at 11.6 g/dl, 83% of patients were transfusion free, and 73% of patients had normalized levels of lactate dehydrogenase (LDH). Long-term safety data were consistent with previous clinical study results, with no thrombotic events or meningitis infections reported. The most common adverse events (AEs) were hemolysis (17%) and injection site reactions (11%).
Presentation details include:
Long-Term Safety and Efficacy of Pegcetacoplan Treatment in Adults with Paroxysmal Nocturnal Hemoglobinuria- 1248 -
About the Long-Term Efficacy and Safety Extension (APL2-307) Study
The APL2-307 study was a nonrandomized, open-label, multicenter Phase 3 extension study of 137 adults with paroxysmal nocturnal hemoglobinuria (PNH) who completed previous EMPAVELI/Aspaveli (pegcetacoplan) Phase 1 (PHAROAH, PADDOCK), Phase 2 (PALOMINO), and Phase 3 (PEGASUS,
About EMPAVELI/Aspaveli (pegcetacoplan)
EMPAVELI/Aspaveli (pegcetacoplan) is a targeted C3 therapy designed to regulate excessive activation of the complement cascade, part of the body's immune system, which can lead to the onset and progression of many serious diseases. It is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) in
About Paroxysmal Nocturnal Hemoglobinuria (PNH)
PNH is a rare, chronic, life-threatening blood disorder characterized by the destruction of oxygen-carrying red blood cells through extravascular and intravascular hemolysis. Persistently low hemoglobin can result in frequent transfusions and debilitating symptoms such as severe fatigue, hemoglobinuria and difficulty breathing (dyspnea).
About the Apellis and Sobi Collaboration
Apellis and Sobi have global co-development rights for systemic pegcetacoplan. Sobi has exclusive ex-
About Apellis
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