Pharmacological Innovations in Familial Hypercholesterolemia Treatment

Abstract

Familial Hypercholesterolemia (FH) represents a significant global health challenge, characterized by elevated low-density lipoprotein cholesterol (LDL-C) levels and an increased risk of atherosclerotic cardiovascular disease (ASCVD). Traditional management strategies, including statins and lifestyle modifications, are often insufficient to achieve optimal LDL-C targets, necessitating the exploration of advanced pharmacological approaches. This review highlights the critical role of pharmacology in mitigating ASCVD risks associated with FH, focusing on the efficacy and safety of current therapies such as statins, PCSK9 inhibitors, and novel agents targeting ANGPTL3 and microsomal triglyceride transfer protein. Furthermore, the potential of emerging modalities, including RNA interference therapies, gene editing, and cholesterol metabolism vaccines, is explored. Advances in risk stratification, incorporating genetic testing and imaging techniques, complement pharmacological innovations by enabling personalized care. These developments underscore the necessity of integrated, multidisciplinary approaches to FH management, emphasizing the promise of pharmacological breakthroughs in addressing unmet clinical needs.