Genome-based cholesterol drug boosts heart health

Juan Gaertner/Science Photo Library
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%.
Worth any price?
Now that the data on evolocumab are in, some health-care payers such as insurance companies and government programmes might be more willing to shoulder the treatment’s steep cost. But any new cholesterol drug faces stiff competition from cheaper statins, which have been used to control LDL levels for decades.

Some analysts say that demonstrating a statistically significant heart-health benefit would not be enough to ensure the PCSK9 drug’s status as the next big thing. “The more important hurdle is the one that payers have imposed restricting access to these medicines,” wrote analysts at the investment bank Leerink Partners in New York City, in a report released 15 March.
To cross that threshold, Leerink’s analysts estimated that evolocumab would need to reduce cardiovascular risks by 25% or more.

Overall, the risk reduction was less than what might have been expected based on how much evolocumab reduces the amount of LDL cholesterol in the body, says Krumholz. But the evidence of a benefit is strong enough that he will discuss the drug as an option with his patients, he adds.

Source: Nature

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