The European Medicine Agency (EMA) approved the use of PCSK9 inhibitors in patients affected by familial hypercholesterolemia (FH), non-familial hypercholesterolemia, or mixed dyslipidaemias as add-on therapy in patients unable to reach the LDL-C target despite high-intensity lipid-lowering treatment (LLT) (or non-statin LLT in case of statin intolerance). The gene discussed is PCSK9; the disease is familial hypercholesterolemia.