Results of Pegcetacoplan (APL-2) trial

A recently published study from Leeds, England, investigates the efficacy of the new drug pegcetacoplan for Paroxysmal Nocturnal Haemoglobinuria (PNH). Pegcetacoplan is not yet approved for PNH therapy in Europe, but approval is currently being considered by the European Medicines Agency (EMA). For this reason, we would like to briefly introduce the drug and the study to you and first take a brief look at the disorder of blood formation underlying PNH in order to understand how pegcetacoplan works:

Normally, certain proteins are found on the surface of the red blood cells (erythrocytes) that transport oxygen through our bodies. Among other things, these regulate the immune system and prevent the erythrocytes from being broken down prematurely as they circulate through the body. In PNH,READ MORE