Federico Grossi, MD, Chief Medical Officer at Apellis Pharmaceuticals, discusses the results of the PEGASUS study, a phase 3 clinical trial testing pegcetacoplan vs eculizumab in patients with paroxysmal nocturnal hemoglobinuria (PNH).

PNH is a rare, life-threatening blood disorder characterized by hemolysis. Persistently low hemoglobin levels can result in severe fatigue and difficulty breathing. Additionally, individuals with PNH are susceptible to thrombotic events and many have some degree of bone marrow dysfunction. 

The latest data from the PEGASUS study shows great promise for the PNH community. In the phase 3 study, there was a significant improvement in hemoglobin levels and patient quality of life in patients receiving pegcetacoplan compared to eculizumab. Previous research shows that even on eculizumab – the standard drug for PNH treatment – 70% of patients still have low hemoglobin levels and 36% require at least one blood transfusion every year. PNH patients, when treated with pegcetacoplan, compared to eculizumab, had more than a 50% increase in hemoglobin levels. 

For more information about PNH, and other rare blood disorders, visit checkrare.com/diseases/hematologic-disorders/.


  • Sign up
Lost your password? Please enter your username or email address. You will receive a link to create a new password via email.
We do not share your personal details with anyone.