An experimental drug in development by Amgen has been shown to drastically reduce bad cholesterol in people who are genetically more likely to have high cholesterol.
The drug at the center of the research is evolocumab, a monoclonal antibody that inhibits a protein known to impair the liver’s ability to clear bad (LDL) cholesterol from the blood. In a recent phase 3 trial, Amgen researchers evaluated the drug in patients with a rare genetic disorder called hypercholesterolemia (HoFH). The condition is associated with dangerously elevated LDL levels with symptoms typically beginning at an early age.
The study consisted of 49 patients with HoFH aged 12 to 17 who were on a regular regimen of statin drugs and other medication to lower lipids. The group was randomized into two groups – those who received evolocumab and those who were given a placebo.
Amgen reports that the percent reduction in bad cholesterol among the evolocumab group was “clinically meaningful and statistically significant.” Company representatives say the drug represents a potential new treatment option for patients with HoFH. According to a Reuters report, other pharmaceutical companies like Pfizer are also working toward the development of a similar drug.
HoFH is a serious condition that significantly raises the risk of developing coronary artery disease. This occurs when excessive cholesterol builds up in the walls of arteries that carry blood to the heart. Over time, these pathways become narrowed, restricting blood flow. The National Institutes of Health reports that about one in 500 people suffer from the disease.