Current, novel and emerging lipid-lowering therapies in pediatric familial hypercholesterolemia

PURPOSE OF REVIEW: Over the past decade, pharmacological treatment options for children with familial hypercholesterolemia (FH) have expanded considerably. The recent European Atherosclerosis Society consensus recommends more stringent LDL cholesterol (LDL-C) targets than previously advocated. Consequently, more children with FH are expected to become eligible for novel therapeutic options. This review summarizes established and emerging lipid-lowering therapies (LLT) evaluated in children, focusing on recent and ongoing clinical trials. RECENT FINDINGS: Conventional LLT remains the cornerstone of FH treatment, and proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors are approved for pediatric use and achieve significant LDL-C reductions; however, injectable administration and high costs limit their widespread use. Novel oral PCSK9 inhibitors are currently under investigation in clinical trials, and fixed-dose combinations may offer a more practical and cost-effective approach. Additionally, angiopoietin-like protein 3 inhibitors and microsomal triglyceride transfer protein inhibitors lower LDL-C in dependently of LDL receptor activity, making them particularly suitable for homozygous FH patients. SUMMARY: The management of FH is entering a new therapeutic era. As therapeutic options expand and agents with greater LDL-C reductions become available, personalized treatment strategies will become increasingly important to achieve LDL-C targets and may ultimately shift from stepwise intensification approaches toward early intensive treatment.