Health Canada approves Empaveli for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria,- SOBI
Sobi North America, the North American affiliate of Swedish Orphan Biovitrum AB (Sobi) 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. 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. 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.
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