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ACC: New Drug Shows Promise in Rare Cholesterol Disorder

Research finds mipomersen lowers LDL in people with homozygous familial hypercholesterolemia

MONDAY, March 15 (HealthDay News) -- In individuals with homozygous familial hypercholesterolemia already undergoing treatment with lipid-lowering drugs, inhibition of apolipoprotein B synthesis by a new drug, mipomersen, may be effective in reducing low-density lipoprotein (LDL) cholesterol, according to a study published online March 14 in The Lancet to coincide with the Annual Scientific Session of the American College of Cardiology, held from March 14 to 16 in Atlanta.

Frederick J. Raal, M.D., of the University of the Witwatersrand in Johannesburg, South Africa, and colleagues randomized 51 patients, 12 years of age and older, to mipomersen 200 mg subcutaneously weekly or placebo for 26 weeks.

The researchers found that the percentage change in LDL cholesterol was significantly higher with mipomersen than with placebo (−24.7 versus −3.3 percent). In terms of adverse events, injection site reactions were more common in the mipomersen group than the placebo group (76 versus 24 percent). In addition, 12 percent of patients in the mipomersen group experienced increases in alanine aminotransferase concentrations of three times or more the upper limit of normal compared to none in the placebo group.

"Inhibition of apolipoprotein B synthesis by mipomersen represents a novel, effective therapy to reduce LDL cholesterol concentrations in patients with homozygous familial hypercholesterolemia who are already receiving lipid-lowering drugs, including high-dose statins," the authors conclude.

The study was supported by ISIS Pharmaceuticals and Genzyme Corporation. Several authors reported financial relationships with these and other pharmaceutical and biotechnology companies.

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