Genome-based cholesterol drug boosts heart health

Mar 21, 2017

By Heidi Ledford

For years, medical researchers have hoped that a burgeoning class of cholesterol drugs targeting a protein called PCSK9 could be the next generation of blockbuster treatments. Now, a large clinical trial has demonstrated that this approach can lower the risk of heart disease. But it’s still unclear whether these drugs — which attempt to mimic a beneficial genetic mutation — will be the breakthrough that scientists and pharmaceutical companies had imagined.

The results, published in the New England Journal of Medicine1 and presented at the American College of Cardiology conference in Washington DC on 17 March, show that a drug called evolocumab (Repatha) reduced the risk of cardiovascular death, heart attack and stroke by about 20% in patients who were already taking other cholesterol-controlling drugs called statins. This reduction in risk is roughly the same magnitude as patients might see from taking statins alone. On another measure that also included hospitalizations for conditions that cause reduced blood flow to the heart, evolocumab reduced the risk by 15%.

The US Food and Drug Administration (FDA) approved evolocumab in 2015 for use in some patients with high cholesterol, based on data showing that the drug could lower levels of ‘bad’ low-density lipoprotein (LDL) cholesterol circulating in the blood by approximately 60%2. But researchers didn’t have evidence then that the drug could also protect against heart attacks or strokes.

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