Treatment of heterozygous familial hypercholesterolemia: what does the future hold?

ABSTRACT

Introduction

Heterozygous familial hypercholesterolemia (heFH) is a common metabolic disease associated with increased cardiovascular risk. Despite treatment with the currently available lipid-lowering agents (statins, ezetimibe, and proprotein convertase subtilisin/kexin type 9 inhibitors), a substantial proportion of patients with heFH does not achieve low-density lipoprotein cholesterol (LDL-C) targets.

Areas covered

The PubMed database was reviewed for relevant papers published up to August 2020. The safety and efficacy of novel agents, namely inclisiran and bempedoic acid, that lower LDL-C levels and might be useful in the management of patients with heFH are discussed.

Expert opinion

The prolonged lipid-lowering effect of inclisiran might improve adherence to treatment in patients with heFH. Bempedoic acid provides additional reductions in LDL-C levels in patients on high-intensity statin treatment; oral administration of this agent might be attractive to some patients. However, it is important to evaluate the effects of these agents on cardiovascular morbidity before they are incorporated in the management of heFH. The cost/benefit of treatment should also be considered, given the increasing complexity of lipid-lowering treatment.

KEYWORDS:

• Bempedoic acid
• familial hypercholesterolemia
• inclisiran

Article highlights

• Several new agents are under development for the management of patients with heterozygous familial hypercholesterolemia (heFH)

• Inclisiran yields substantial and prolonged reductions in low-density lipoprotein cholesterol (LDL-C) levels

• Bempedoic acid results in more moderate reductions in LDL-C levels but is orally administered

• For all these new agents, the effect on cardiovascular events is unknown and cost-benefit ratio should be evaluated

Declaration of interest

The authors have no relevant affiliations or financial involvement with any organization or entity with a financial interest in or financial conflict with the subject matter or materials discussed in the manuscript. This includes employment, consultancies, honoraria, stock ownership or options, expert testimony, grants or patents received or pending, or royalties.

Reviewer disclosures

Peer reviewers on this manuscript have no relevant financial or other relationships to disclose.

Additional information

Funding

This paper was not funded.