Bempedoic acid, sold under brand names like Nexletol and in combination with ezetimibe as Nexlizet, is an oral, non-statin medication used to manage high levels of low-density lipoprotein cholesterol (LDL-C), or “bad cholesterol.” It is a first-in-class treatment that reduces cholesterol levels by working differently from traditional statins within the liver. This medication is generally prescribed daily alongside dietary changes and other lipid-lowering therapies for patients who require further LDL-C reduction to lower their risk of cardiovascular events.
The Target Patient Profile
This medication is typically prescribed for two patient groups whose cholesterol levels remain elevated despite existing treatment efforts. The first group includes individuals with established atherosclerotic cardiovascular disease (ASCVD) who need further LDL-C reduction to meet treatment goals. This group has already experienced a heart attack, stroke, or other event caused by arterial plaque buildup.
The second patient population consists of those who cannot tolerate statin therapy due to muscle-related side effects, a condition known as statin intolerance. Bempedoic acid offers a valuable alternative because its mechanism of action is liver-specific and does not activate in skeletal muscle, avoiding statin-related muscle discomfort. It is also indicated for adults with primary hyperlipidemia, including heterozygous familial hypercholesterolemia (HeFH). For these patients, it can be used as an add-on therapy or as a monotherapy when statins are not an option.
Understanding Real-World Side Effects and Tolerability
The patient experience with bempedoic acid is generally favorable, especially when compared to the muscle pain commonly associated with high-dose statins. This improved tolerability stems from the drug’s unique activation process, which occurs exclusively in the liver, sparing the skeletal muscles. Despite this muscle-sparing design, patients have still reported some muscle discomfort (myalgia) and joint pain (arthralgia) in real-world settings.
Joint pain, in particular, has been one of the more common complaints reported in clinical trials and subsequent patient feedback. Other frequently noted side effects include symptoms similar to a common cold, such as upper respiratory tract infections, and abdominal discomfort. While many patients find the side effects manageable, some discontinue the medication because of persistent symptoms.
A more serious, though rare, concern is the potential for tendon rupture or injury, which has been reported in about 0.5% of patients in clinical trials. This risk is slightly higher for people over 60, those with kidney problems, or individuals taking specific medications like corticosteroids or fluoroquinolones. Patients should immediately report any sudden pain, swelling, or a snapping sound in their joints or tendons. Bempedoic acid can also cause an increase in blood uric acid levels, which may lead to gout flares in susceptible individuals.
Practical Medication Logistics
Bempedoic acid is prescribed as an oral tablet, typically taken once a day at a fixed dose of 180 mg, and can be taken with or without food. Compliance with this simple, once-daily regimen is generally high, which is an important factor in maintaining long-term cholesterol control.
Patients using bempedoic acid require periodic laboratory monitoring to assess for potential metabolic changes. Clinicians must regularly check blood uric acid levels, particularly in those with a history of gout, as the drug’s effect on renal transporters can lead to hyperuricemia. Monitoring liver enzymes is also standard practice.
A specific drug interaction involves certain statins. Co-administration of bempedoic acid with simvastatin doses greater than 20 mg or pravastatin doses greater than 40 mg is not recommended. This restriction exists because bempedoic acid can increase the concentration of these specific statins in the blood, raising the risk of muscle-related side effects and potential myopathy.
Cost and Insurance Considerations
As a newer, brand-name medication, bempedoic acid has a substantially higher list price compared to generic statins, significantly impacting patient access and affordability. The retail cost for a 30-day supply of bempedoic acid can be around $420 without insurance coverage. This often translates to significant out-of-pocket costs for the patient, depending on their insurance plan design.
Due to the cost and its positioning as a non-first-line therapy, insurance companies almost universally require a prior authorization (PA) before they will cover the prescription. This process means the prescriber must submit documentation proving the patient meets the strict criteria for the drug, such as documented statin intolerance or failure to reach LDL-C goals on other therapies. Initial rejection rates for prescriptions can be high, with some analyses showing initial denials for over 40% of prescriptions.
Even when a prescription is approved, the high patient copay can lead to a significant rate of prescription abandonment, where the patient opts not to fill the medication. Patients are advised to check their coverage details and any financial assistance programs before starting treatment to avoid unexpected costs.