New approaches to address dyslipidemia

Although lipid-lowering treatment with statins, ezetimibe, and PCSK9 inhibitors is a very successful strategy to prevent cardiovascular events, there is a need for further drug developments. Not all patients respond sufficiently to the available therapy (very high baseline values, intolerance). Furthermore, patients may be characterized by dyslipidemias not accessible to available drugs such as patients with homozygous familial hypercholesterolemia, chylomicronemia syndrome, or elevated lipoprotein(a). A number of drugs are being developed to close these gaps.

The focus is on new antibodies, antisense oligonucleotides, and small molecules that address different aspects of lipid metabolism. Many of these developments are promising as they decrease LDL-cholesterol and/or non-HDL-cholesterol and/or triglycerides and/or lipoprotein(a) in patients who so far cannot be treated sufficiently. These drugs are currently in different stages of development and being tested in clinical trials.

Some of the new lipid-lowering drugs have a very promising profile. However, eventually phase 3 and outcome trials will be required to prove the usefulness of these compounds in clinical practice. Furthermore, it is unlikely that they will change the primary lipidological approach (statin and ezetimibe) even if they prove successful.