On March 22, 2024, the US Food and Drug Administration authorised the addition of indications for extended LDL-C reduction in both primary and secondary prevention of cardiovascular (CV) risk to the wide labels of bempedoic acid and bempedoic acid with ezetimibe tablets.

Bempedoic acid for treating CVD

The enlarged indications include a therapeutic indication for primary hyperlipidemia, which may be treated with bempedoic acid alone or in conjunction with ezetimibe as monotherapy or in conjunction with statin medications. The two oral, once-daily medicines now have extended indications, making them the only non-statin treatments that decrease LDL-C and are recommended for main CVD prevention. The non-statin drugs' prospective patient base has now expanded to over 70 million adult US citizens who either have hyperlipidemia-related CVD or are at risk for it.

CLEAR outcomes 

1. The indications for CV risk reduction that have been widely expected are based on data from the CLEAR Outcomes clinical trial, which evaluated bempedoic acid's effects on CV outcomes in a cohort of 14,000 individuals with high-risk or diagnosed CVD who were either statin-intolerant or refused to take the medication.
2. A median of 3.4 years was spent tracking the participants. Researchers documented in The New England Journal of Medicine that participants who were treated with bempedoic acid observed a 15% reduction in the relative risk of MACE-3 (cardiovascular mortality, nonfatal stroke, or nonfatal myocardial infarction) along with a 27% decrease for MI and 19% decrease for coronary revascularization.
3. Furthermore, those who got bempedoic acid did not exhibit an increase in glucose in comparison to those who received a placebo. The researchers also documented decreases in LDL-C and hsCRP of 20% and 22%, respectively.
4. The impact of bempedoic acid vs placebo on the overall incidence of CV events during the study was evaluated in a prespecified analysis of CLEAR Outcomes published in January 2024.
5. The results showed that treatment with the drug was associated with relative risk reductions of 20% for 4-point MACE, 17% for 3-point MACE, 31% for MI, and 22% for coronary revascularization.
6. Bempedoic acid and bempedoic acid with ezetimibe are once-daily oral drugs that lower cardiovascular risk and LDL-C levels without causing the negative effects associated with statins.
7. Additionally, they are the first oral non-statin LDL-C lowering medications that have received FDA approval to lessen the risk of cardiovascular events in individuals receiving primary and secondary preventive care.
8. This dramatically changed profile is revolutionary for both patients and healthcare professionals.

Concise background

1. The FDA first authorised bempedoic acid in February 2020 as an adjuvant medication to maximally tolerated statin therapy for the treatment of existing atherosclerotic cardiovascular disease or heterozygous familial hypercholesterolemia.
2. Additionally, at the time, the label said that bempedoic acid's impact on CV morbidity and death was unknown.
3. To qualify for the currently authorised populations, eligible persons must treat their primary hyperlipidemia, according to the first label expansion that was revised in December 2023.
4. Significantly, the label's need for maximally tolerated statin medication as a precondition for bempedoic acid treatment was eliminated, as was the use restriction mentioning the uncertain impact on cardiovascular morbidity and mortality.

Disclaimer- The views and opinions expressed in this article are those of the author and do not necessarily reflect the official policy or position of M3 India.

About the author of this article: Dr Monish Raut is a practising super specialist from New Delhi.