In , the authors demonstrated the safety and efficacy of the drug in a large, placebo-controlled trial.
For one year, 2,230 patients with atherosclerosis, high cholesterol, or both took part in the trial, with two-thirds of them taking an oral dose of bempedoic acid and the remainder using a placebo.
Those using bempedoic acid lowered their average LDL cholesterol level by an average of 18 percent compared with the placebo group.
Researchers also found the drug to be safe and well-tolerated. During that time period, both groups had similar amounts of adverse health incidents — including heart attacks and death. That is, bempedoic acid did not increase the health risks for those taking it.
It is the first study to measure these outcomes.
“Bempedoic acid is an exciting new medication used to lower cholesterol,” said , director of cardiovascular health and lipidology at Northwell Health’s Sandra Atlas Bass Heart Hospital in Manhasset, New York.
“Based on this study bempedoic acid can safely be added to statin therapy for those patients who require further LDL cholesterol reduction,” said Mintz, who is not affiliated with the study.
The demonstrates a biological proof of concept for how bempedoic acid works in the body. Using biologic data and biomarkers from over a half a million people, researchers modeled the likely effects of the treatment over a longer period of time. The study reinforced the conclusion that the effects of the drug would reduce cardiovascular disease without serious adverse health outcomes.
Bempedoic acid works by blocking the production of ATP citrate lyase, one of the key enzymes in the production of cholesterol.
Bempedoic acid actually works , just further “upstream” in the process. Statins work by inhibiting the body’s production of an enzyme called HMG-CoA reductase, another enzyme used to make cholesterol.
By inhibiting this enzyme, the liver produces less cholesterol, thereby lowering cholesterol levels.
“Many patients do not get enough LDL lowering for their level of risk… So this is a huge area of unmet need as the only alternative is to leave people at higher risk,” said , professor of public health in the Ddartment of public health and primary care at Imperial College London, an author on both publications.
Statins are some of the most prescribed drugs in the world, but they don’t work for everyone. Even when they do work, sometimes their effects aren’t sufficient. They can also cause The most common include digestive problems, fatigue, cramps, and muscle pain (myalgias).
A found that over 10 percent of people taking high-dose statins had muscle pain. These side effects, depending on severity, can lead some to stop taking their medication.
“Reasons for stopping vary sometimes: muscle aches, lethargy, and nonspecific reasons [such as] ‘just don’t feel right.’ Not all of these are proven to be causal but if a patient feels something is related to the drug and there is a lot of adverse publicity [about] statins they might not take it,” said Ray.
A different mechanism of action could be beneficial for these individuals.
“This new medication may have a role in lowering cholesterol in patients that cannot tolerate statin medications at all or cannot tolerate the dosages needed to get to cholesterol goal,” said Mintz. “The mechanism to explain less muscle aches with bempedoic acid is that muscle cells do not have the enzyme needed to activate bempedoic acid and it would not be active there.
Cost is also an issue.
Generics are available for statins, which can make them a cheap and accessible option for many people. However, novel cholesterol-lowering drugs known as PCSK9 inhibitors (alirocumab and evolucumab), are significantly more expensive,
For those who can’t use statins, PCSK9 inhibitors could be out of reach. Both Ray and Mintz project that bempedoic acid treatment will likely fall between the two in terms of cost, potentially giving people with high cholesterol another option.
According to , Esperion Therapeutics thinks this drug could cost around $10 a day or around $4,000 a year, although that number has yet to be verified by experts outside the company.
“Both studies are reassuring of the safety and benefit of bempedoic acid and provide hope for patients that cannot take statins, or cannot tolerate enough statin to get their cholesterol low enough. This will be another effective tool in the fight against heart disease,” said Mintz.
In a pair of articles published today in the New England Journal of Medicine, researchers tout the potential of bempedoic acid as a powerful new tool in the fight against heart disease. The studies were funding by the pharmaceutical company that is looking to release the drug in the United States and Europe.
Bempedoic acid may help lower cholesterol levels in some people according to the research. The medication could help people with high cholesterol who do not respond to statins or who can’t afford expensive drugs known as PCSK9 inhibitors.