Genzyme and Isis Pharmaceuticals announced that their Phase 3 study of mipomersen in patients with homozygous familial hypercholesterolemia (hoFH) met its primary endpoint, with a 25 percent reduction in LDL cholesterol after 26 weeks of treatment, vs. 3 percent for placebo (p<0.001). This study also met each of its three secondary endpoints of reduction in apolipoprotein B, total cholesterol and non-HDL cholesterol (all p<0.001). The trial was a randomized, double-blind, placebo-controlled study that enrolled 51 hoFH patients, who were aged ≥12 years old. Patients were randomized 2:1 to receive a 200 mg dose of mipomersen or placebo via weekly injections for 26 weeks.
Mipomersen is an apo-B synthesis inhibitor that reduces LDL-C by preventing the formation of atherogenic lipoproteins.
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