In short
Bempedoic acid is an innovative drug being studied in clinical trials for its potential to lower cholesterol levels and reduce cardiovascular risk. This article explores the use of bempedoic acid in various clinical trials, examining its efficacy, safety, and potential benefits for patients with hyperlipidemia and other cardiovascular risk factors.
At a glance
- Primary Use
- Lowering LDL cholesterol in patients with hyperlipidemia
- Mechanism of Action
- Inhibition of ATP-citrate lyase enzyme in the liver
- Efficacy
- Significant reduction in LDL-C levels (17-18% vs placebo in some trials)
- Safety Profile
- Generally well-tolerated; long-term safety data still being collected
- Combination Therapy
- Studied with statins, ezetimibe, and as monotherapy
- Target Populations
- Patients with hyperlipidemia, ASCVD, statin intolerance, HeFH
- Additional Benefits
- Potential improvements in inflammation markers and glycemic parameters
- Ongoing Research
- Long-term cardiovascular outcomes, use in specific populations (e.g., pediatric HeFH)
What is Bempedoic Acid?
Bempedoic acid is a medication used to lower cholesterol levels in the blood. It's also known by the brand names Nexletol (when used alone) and Nexlizet (when combined with another cholesterol-lowering drug called ezetimibe). Bempedoic acid is a new type of cholesterol-lowering drug that works differently from other medications like statins.
How Does It Work?
Bempedoic acid works by blocking an enzyme in the body called ATP-citrate lyase (ACL). This enzyme is involved in the production of cholesterol in the liver. By inhibiting ACL, bempedoic acid reduces the amount of cholesterol your body makes. This leads to lower levels of LDL-C (often called "bad" cholesterol) in your blood. It also helps your liver remove more LDL-C from your bloodstream.
What Conditions Does Bempedoic Acid Treat?
Bempedoic acid is primarily used to treat the following conditions:
- Hypercholesterolemia: This is a condition where you have high levels of cholesterol in your blood.
- Atherosclerotic Cardiovascular Disease (ASCVD): This refers to the buildup of plaque in your arteries, which can lead to heart attacks and strokes.
- Heterozygous Familial Hypercholesterolemia (HeFH): This is an inherited condition that causes very high cholesterol levels from birth.
Bempedoic acid is usually prescribed for people who can't take statins (another type of cholesterol-lowering drug) due to side effects, or for those who need additional cholesterol lowering even when taking the highest tolerated dose of statins.
How Effective is Bempedoic Acid?
Clinical trials have shown that bempedoic acid can significantly lower LDL-C levels. When added to other cholesterol-lowering treatments, it can provide additional benefits:
- When added to maximally tolerated statin therapy, bempedoic acid lowered LDL-C by about 18% more than placebo after 12 weeks of treatment.
- In patients who can't take statins, bempedoic acid lowered LDL-C by about 28% compared to placebo after 12 weeks.
- When combined with ezetimibe (another cholesterol-lowering drug), the combination lowered LDL-C by about 38% more than placebo.
In addition to lowering LDL-C, bempedoic acid also reduces other markers of cardiovascular risk, such as non-HDL cholesterol, total cholesterol, and apolipoprotein B.
Potential Side Effects
Like all medications, bempedoic acid can cause side effects. Common side effects reported in clinical trials include:
- Upper respiratory tract infections
- Muscle spasms
- Back pain
- Abdominal pain or discomfort
- Increased levels of uric acid in the blood
In some cases, bempedoic acid may cause increases in liver enzymes or creatinine (a marker of kidney function). Your doctor will monitor these with blood tests.
Use in Special Populations
Bempedoic acid is being studied in various populations:
- Children and Adolescents: A study is underway to evaluate the use of bempedoic acid in children and adolescents (ages 6-17) with heterozygous familial hypercholesterolemia.
- Pregnant Women: A pregnancy surveillance program is in place to assess the safety of bempedoic acid in pregnant women and their babies.
- People with HIV: A study is investigating the effects of bempedoic acid on cardiovascular risk in people living with HIV.
Ongoing Research
Research on bempedoic acid is ongoing. Some areas of current investigation include:
- Long-term safety and effectiveness
- Use in combination with other cholesterol-lowering drugs
- Effects on liver fat in people with non-alcoholic fatty liver disease and type 2 diabetes
These ongoing studies will help researchers and doctors better understand how to use bempedoic acid to improve heart health in different groups of patients.