Paroxysmal nocturnal hemoglobinuria Paroxysmal nocturnal hemoglobinuria: (par-uk-SIZ-muhl nok-TURN-uhl hee-muh-gloe-buh-NYOOR-ee-uh) A rare and serious blood disease that causes red blood cells to break apart. Paroxysmal means sudden and irregular. Nocturnal means at night. Hemoglobinuria means hemoglobin in the urine. Hemoglobin is the red part of red blood cells. A… (PNH) is a rare, life-threatening acquired blood disease characterized by chronic complement-mediated hemolysis hemolysis: (hi-MOL-uh-suss) The destruction of red blood cells. and thrombosis thrombosis: (throm-BOE-suss) A blood clot (thrombus) that develops and attaches to a blood vessel. . On May 14, 2021, the US FDA approved a new targeted C3 therapy Empaveli (pegcetacoplan), once called APL-2, for use in adult PNH. This review aims to review the pharmacological properties, clinical safety and efficacy of pegcetacoplan pegcetacoplan: EMPAVELI® is the first PNH treatment that binds to complement protein C3. It was approved by the Food and Drug Administration in May 2021 for treating adult patients with paroxysmal nocturnal hemoglobinuria (PNH). EMPAVELI is given skin (subcutaneously) by using the Empaveli injector or with an… , and provides comprehensive drug information about pegcetacoplan. Pegcetacoplan is a pegylated peptide that targets the proximal complement protein C3. Pegcetacoplan plays a role in the complement cascade that controls C3b-mediated extravascular hemolysis and terminal complement-mediated intravascular hemolysis. Early pharmacokinetic and pharmacodynamic trials proved that pegcetacoplan had good tolerability and acceptable safety, while reducing complement activity. The pivotal phase III trial PEGASUS of PNH patients with a suboptimal response to eculizumab eculizumab: Eculizumab (Soliris ®) is given as an IV into a vein at the doctor’s office or at a special center. The procedure usually takes about 35 minutes. You will probably get an IV once a week for the first 4 weeks. Starting in the 5th week, you will get a slightly higher dose of Soliris every 2 weeks. … reported that pegcetacoplan improved hemoglobin hemoglobin: A protein in the red blood cells. Hemoglobin picks up oxygen in the lungs and brings it to cells in all parts of the body. levels better than eculizumab (p < 0.001). Compared with eculizumab, patients who received pegcetacoplan had a higher chance of not requiring a blood transfusion blood transfusion: A procedure in which whole blood or one of its components is given to a person through an intravenous (IV) line into the bloodstream. A red blood cell transfusion or a platelet transfuson can help some patients with low blood counts. within 16 weeks (85% vs. 15%, p < 0.001). The safety of pegcetacoplan was similar to that of eculizumab. Adverse events such as injection site reactions and diarrhea occurred frequently following pegcetacoplan administration. The prescription recommended dosage is 1080 mg, subcutaneously infusion twice a week.
Keywords: APL-2; C3; Empaveli; Hemolysis; Paroxysmal nocturnal hemoglobinuria; Pegcetacoplan.