Abstract
Twelve years ago, a discovery was made of a new protein termed as proprotein convertase subtilisin/kexin type 9 (PCSK9), which significantly affects the levels of serum LDL-cholesterol. In a very short period of time, research led to preparing several options how to therapeutically affect PCSK9.
Among the most advanced is the development of specific monoclonal antibodies blocking this protein. As a result, the number of recirculating LDL-receptors on the hepatocyte surface is increased, leading to a reduction in serum LDL-cholesterol (and other atherogenic lipids) by 50-70 % beyond the level achievable with other available therapies.
The safety and efficacy of PCSK9 inhibitors is being tested in an extensive research programme involving several manufacturers. This novel therapeutic option represents hope for difficult-to-treat patients with familial hypercholesterolaemia or statin intolerance and, in the future, even for those with very high and high cardiovascular risk failing to reach target LDL-cholesterol levels with currently available therapies.