EMA has recommended granting a marketing authorisation in the European Union (EU) for Redemplo (plozasiran) to treat adults with familial chylomicronaemia syndrome (FCS).

FCS is a rare inherited disease that prevents the body from breaking down lipids (fats). People with this condition have extremely high levels of triglycerides in their blood. This causes a range of symptoms, including severe abdominal pain, potentially fatal attacks of acute pancreatitis, hepatosplenomegaly (enlargement of liver and spleen), diabetes, lack of concentration, memory loss and fat-filled spots on the skin (called xanthomas).

People with FCS must strictly limit their fat intake through diet. However, this is not always feasible nor sufficiently effective to reduce the level of triglycerides and prevent pancreatitis. Traditional lipid-lowering medications have minimal impact when it comes to reducing triglyceride levels in patients with FCS.

The active substance in Redemplo, plozasiran, is a…