Monica Fay, PharmD, Senior Vice President of Global Medical Affairs at Apellis Pharmaceuticals, provides an overview of paroxysmal nocturnal hemoglobinuria (PNH).
As Dr. Fay explains, 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.
In May 2021, the U.S. Food and Drug Administration (FDA) approved pegcetacoplan to treat PNH. Even more recently, data from the phase 3 PRINCE study (NCT04085601), demonstrating that pegcetacoplan sustains normalization of clinical measures in a broad PNH patient population, were presented at The American Society of Hematology Meeting & Exposition (ASH 2021).
To learn more about PNH and other rare blood disorders, visit checkrare.com/diseases/hematologic-disorders/.