Ricotta DN et al. – Clinical outcome studies looking at cardiovascular disease end points still need to be done.

• 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.