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Bempedoic acid and inclisiran for patients with heterozygous familial hypercholesterolemia and for secondary prevention of ASCVD: Effectiveness and value : final report
Bempedoic acid and inclisiran for patients with heterozygous familial hypercholesterolemia and for secondary prevention of ASCVD: Effectiveness and value : final report
- Collection:
- Health Policy and Services Research
- Author(s):
- Lin, Grace A., author
Jih, Jane, author
Agboola, Foluso, author
Peason, Steven D., author
Kazi, Dhruv S., author - Contributor(s):
- Institute for Clinical and Economic Review, issuing body.
Midwest Comparative Effectiveness Public Advisory Council, issuing body. - Publication:
- [Boston, Massachusetts] : Institute for Clinical and Economic Review, March 2, 2021
- Language(s):
- English
- Format:
- Text
- Subject(s):
- Anticholesteremic Agents -- therapeutic use
Atherosclerosis -- prevention & control
ATP Citrate (pro-S)-Lyase -- antagonists & inhibitors
Dicarboxylic Acids -- therapeutic use
Fatty Acids
Hyperlipoproteinemia Type II -- drug therapy
RNA, Small Interfering
Secondary Prevention
Treatment Outcome
Drug Therapy Combination
Ezetimibe -- therapeutic use
United States - Genre(s):
- Technical Report
- Abstract:
- Atherosclerotic cardiovascular disease (ASCVD) encompasses a set of common, complex, and burdensome conditions with coronary artery disease, peripheral artery disease, and cerebrovascular disease as the three most prevalent types. Almost 1 in 10 people are estimated to have some form of ASCVD, and ASCVD remains the leading cause of death in the United States. There are significant disparities in ASCVD burden by race and sex, with Hispanic and non-Hispanic Black men and women at higher risk of death compared with White men. The financial cost of the disease is staggering, with total costs expected to reach $1.1 trillion by 2035. Risk factors for ASCVD include diabetes mellitus, hypertension, obesity, smoking, and elevated levels of cholesterol, particularly low-density lipoprotein cholesterol (LDL-C). A genetic disorder of cholesterol metabolism, familial hypercholesterolemia (FH), can lead to severely elevated plasma concentrations of LDL-C, placing patients at higher risk of major adverse cardiac events (MACE) such as myocardial infarctions and strokes earlier in life. Although heterozygous FH (HeFH) is the most common form of FH, affecting approximately 1 in 250 people in the US, the condition is underdiagnosed and undertreated, particularly amongst women, Blacks, and Asians. Treatment of patients with FH and established ASCVD includes risk factor modification such as dietary and lifestyle changes and smoking cessation, medical therapy, and when necessary, percutaneous or surgical revascularization. Because of the association between lipid levels and MACE, medical therapy should include intensive lipid-lowering therapy, with a goal LDL-C reduction of at least 50%. Ideally this should be accomplished with a high dose or maximally tolerated statin, but for patients who continue to have LDL-C levels at or above 70 mg/dL, the addition of ezetimibe is recommended as second-line therapy. Finally, for those patients who continue to have LDL-C levels above 70 mg/dL on statin and ezetimibe, a proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitor can be considered. For patients who have statin-associated side effects (SASE) (also known as statin intolerance)--defined as not able to tolerate moderate to high intensity statin therapy due to side effects--therapy with ezetimibe, PCSK9 inhibitors, and other lipid-lowering therapies may be considered to reach treatment goals. Even with the wide range of aforementioned options for risk factor modification and treatment, patients with HeFH and established ASCVD, who are the focus of this review, remain at high residual risk for further MACE, particularly if LDL-C levels are not adequately controlled. Thus, there is an important public health need for additional treatment options to improve outcomes for patients who remain at higher risk for cardiovascular events. One new lipid-lowering treatment has recently been approved by the FDA: bempedoic acid with or without ezetimibe (Nexlizet and Nexletol, Esperion Therapeutics, Inc.),, an inhibitor of adenosine triphosphate (ATP) citrate lyase that lowers LDL-C by reducing cholesterol synthesis and up-regulating LDL receptors. Another agent currently under evaluation at the FDA is inclisiran (Novartis), a double-stranded small interfering RNA agent targeting and inhibiting hepatic PCSK9 synthesis. These therapies are the focus of this review.
- Copyright:
- Reproduced with permission of the copyright holder. Further use of the material is subject to CC BY license. (More information)
- Extent:
- 1 online resource (1 PDF file (various pagings))
- Illustrations:
- Illustrations
- NLM Unique ID:
- 9918680387906676 (See catalog record)
- Permanent Link:
- http://resource.nlm.nih.gov/9918680387906676