Sobi North America, the North American affiliate of Swedish Orphan Biovitrum AB, announced that Health Canada has approved Empaveli™ (pegcetacoplan), a complement inhibitor, for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) who have an inadequate response to, or are intolerant of, a C5 inhibitor. Empaveli™ is a targeted C3 therapy designed to regulate the activity of the complement cascade, which can lead to the onset and progression of many serious diseases.
PNH is a rare, life-threatening hematologic disorder, acquired genetically, with numerous manifestations. PNH is characterized by the destruction of red blood cells (hemolysis) and release of free hemoglobin that can cause thrombosis and require frequent transfusions. Hemolysis can reduce quality of life by causing debilitating symptoms such as dyspnea, pain, erectile dysfunction and severe fatigue caused by anemia.2-4 The global incidence of PNH is estimated to be 1-1.5 cases per million individuals and is estimated to impact fewer than 1,000 people across Canada.4 Despite reductions in hemolysis with C5 inhibitor treatment, approximately 70 percent of people with PNH treated with C5 inhibitors remain anemic, according to a retrospective study and a cross-sectional study.5,6
“Today’s approval by Health Canada is a significant milestone for those living with PNH across Canada,” said Bob McLay, Vice President, General Manager of Sobi Canada. “With the addition of Empaveli, patients now have the added flexibility of a treatment option that has demonstrated superiority to the previous standard of care and can be self-administered, reducing the need to schedule infusions in a clinic and giving them back the gift of time.”
The approval is based on the results from the head-to-head PEGASUS phase 3 study, which evaluated the efficacy and safety of Empaveli compared to eculizumab at 16 weeks in adults with PNH who had persistent anemia despite treatment with eculizumab. The full safety and efficacy results were published in The New England Journal of Medicine in March 2021.1
“Many of our PNH patients treated with C5 inhibitors remain anemic despite full terminal complement control. Some of them have persistent fatigue, reduced quality of life, and may require ongoing transfusions” said Christopher Patriquin, hematologist at Toronto General Hospital and an investigator on the PEGASUS trial. “Today’s approval represents an important new treatment option for Canadian PNH patients who may have an inadequate response to currently available therapies.”