The role of mipomersen therapy in the treatment of familial hypercholesterolemia

Abstract

Familial hypercholesterolemia (FH) is a genetic disorder that results in elevated LDL levels and is associated with an increased risk of cardiovascular disease. HMG-CoA reductase inhibitors are recommended as first-line treatment for FH, but often multiple lipid-lowering agents are required to achieve desired reductions in LDL-C in this patient population. The purpose of our review is to discuss the role of mipomersen in the treatment of FH. A PubMed search revealed three published studies and three abstracts of Phase III extension studies evaluating mipomersen in the treatment of FH, and one study evaluating the safety and efficacy of mipomersen in statin-intolerant patients at high risk for cardiovascular disease, wherein a little more than half of the enrolled patients were diagnosed with FH. Mipomersen, an antisense single-strand synthetic oligonucleotide, may become a potential treatment option for patients with FH that require either additional lipid-lowering or as an alternative to statins in intol...