Phase 3 study of mipomersen for the treatment of heterozygous familial hypercholesterolemia (heFH)

Genzyme Corp. and Isis Pharmaceuticals announced statistically significant results in their Phase 3 study of mipomersen in patients with heterozygous familial hypercholesterolemia (heFH).  This study was a randomized, double-blind, placebo-controlled trial that enrolled 124 heFH patients, aged 18 and older with LDL-C levels greater than 100 mg/dL. Patients were randomized 2:1 to receive a 200 mg dose of mipomersen or placebo weekly for 26 weeks conducted at 26 sites in both the U.S. and Canada.  This study met its primary endpoint with a highly statistically significant 28 percent reduction in LDL-cholesterol after 26 weeks of treatment with mipomersen, compared with an increase of 5 percent for placebo.

Mipomersen is a first-in-class apo-B synthesis inhibitor currently in late-stage development. It is intended to reduce LDL-C by preventing the formation of atherogenic lipids. It acts by decreasing the production of apo-B, which provides the structural core for all atherogenic lipids, including LDL-C, which carry cholesterol through the bloodstream.

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