[PCSK9 inhibitors : Current clinical relevance]

Hypercholesterolemias are known risk factors for cardiovascular diseases. Although statins have reduced the cardiovascular morbidity and mortality and further therapeutic measures are available, treatment goals are often not achieved. In cases of very high levels of low-density lipoprotein (LDL) cholesterol or of intolerability, the established therapies are often not sufficiently effective or cannot be used in adequate doses. For these high-risk patients further treatment options are required.

The current clinical relevance of the new substance class of proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors for the treatment of hypercholesterolemias is presented on the basis of the available data and the German regulations.

The two PCSK9 inhibitors, evolocumab and alirocumab, were approved in 2015. Data from many different patient groups are available for both substances. The significant reduction of LDL cholesterol of 50-60% and the very good tolerability and safety profile (at placebo level) are shown for both substances. The PCSK9 inhibitors are not as effective only in homozygous familial hypercholesterolemia. The first long-term data and one imaging study raise hope that the endpoint trials will show the expected reduction in cardiovascular events. Long-term trials have to show the long-term safety. In Germany it is legally regulated which patients can be treated by PCSK9 inhibitors and these prerequisites are largely in accordance with clinical practice.

The body of evidence is rapidly increasing thereby facilitating the decision making when PCSK9 inhibitors could be used. The PCSK9 inhibitors will considerably improve the options for optimal treatment of high-risk patients.