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Mipomersen: A Safe and Effective Antisense Therapy Adjunct to Statins in Patients With Hypercholesterolemia

Ricotta, Daniel N. MD*; Frishman, William MD

doi: 10.1097/CRD.0b013e31823424be
New Therapy Update

Mipomersen is an antisense oligonucleotide inhibitor of apolipoprotein (apo) B-100 currently in phase 3 of development for the treatment of hyperlipidemia in patients with a high risk for cardiovascular disease. The drug acts by inhibiting the production of apoB-100, which is the structural core for all atherogenic lipids, including low-density lipoprotein cholesterol (LDL-C). The agent has been shown to produce significant reductions in LDL-C from baseline values compared with placebos. Clinical trials have demonstrated that mipomersen reduces LDL-C up to 44% in patients with familial hypercholesterolemia and patients with significantly elevated LDL despite taking maximum doses of statins. Unlike other medications that target apoB-100, such as microsomal triglyceride transfer proteins, mipomersen does not cause hepatic steatosis or intestinal steatosis and does not affect dietary fat absorption. Adverse side effects encountered with mipomersen include flu-like symptoms, injection site reactions, and elevated liver transaminases. If future studies continue to show such promising results, mipomersen would likely be a viable additional lipid-lowering therapy for patients who are at high cardiovascular risk, intolerant to statins, and/or not at target lipid levels despite maximum doses of statin therapy. Clinical outcome studies looking at cardiovascular disease end points still need to be done.

*Departments of Medicine, Harvard Medical School/Beth Israel Deaconess Medical Center, Boston, MA

New York Medical College/Westchester Medical Center, Valhalla, NY

Disclosure: The authors declare no conflict of interest.

Correspondence: William H. Frishman, MD, Department of Medicine, Munger 263, New York Medical College, Valhalla, NY 10595. E-mail:

© 2012 Lippincott Williams & Wilkins, Inc.