CardioExchange Archive

Frederic J Raal et al. report results in the Lancet of a phase 3 study in patients with homozygous familial hypercholesterolemia. Fifty one subjects on high dose lipid-lowering drugs were randomized (on a 2:1 basis) to either placebo or mipomersen, a novel antisense inhibitor of apolipoprotein B synthesis. Patients on mipomersen had a 24.7% decrease in LDL, compared with a 3.3% decrease with placebo (p=0.0003). However, most patients on mipomersen failed to reach target LDL levels. Increases of alanine aminotransferase of three times or more the upper limit of normal was observed in 4 (12%) of patients on mipomersen but none of the patients on placebo. In an accompanying comment,  Neely and Bassendine said that the drug “is a promising new treatment option for refractory hypercholesterolemia, with the potential to be used in combination with statins in patients with genetically defined familial hypercholesterolemia who have not responded adequately, or as monotherapy in those who are intolerant of lipid-lowering regimens.” Broader use of the drug, however, would require more studies to evaluate the safety of the drug, they wrote.