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The Role of Mipomersen Therapy in the Treatment of Familial Hypercholesterolemia

Roda Plakogiannis, Lisa Cioce, Edward A Fisher, James A Underberg

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 intolerant individuals. Studies have demonstrated that mipomersen significantly reduces LDL-C, ApoB, non-high-density lipoprotein-C, triglycerides and lipoprotein(a) when added to traditional lipid lowering therapy in patients with FH. Safety analysis has shown that mipomersen is well tolerated except for the common finding of injection-site reactions. Further, hepatic steatosis is an ongoing safety concern that warrants future investigation with larger populations and longer study periods. Overall, mipomersen may offer an additional treatment option for patients with FH and has been shown to effectively reduce cholesterol in this population in short-term studies.