PCSK9 inhibitors represent a class of medications that act directly or indirectly on a protein called PCSK9 that modulates the expression of the LDL receptor. Patients with nonsense mutations in the PCSK9 gene have a significantly lower level of LDL-C and a decreased incidence of CHD. In preclinical trials, over-expression of PCSK9 has resulted in increased levels of non-HDL cholesterol. Two recent monoclonal antibody trials that were presented at the American Heart Association (AHA) meeting in 2011 showed that LDL-C was lowered by an average of up to 60%.
