PCSK9 Inhibitors: Uses, most common brand names, and safety information
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The role of lipids (cholesterol) in cardiovascular disease (CVD) was established in clinical studies over 50 years ago. Genetic causes of high cholesterol levels (hyperlipidemia) are relatively common. Familial hypercholesterolemia (FH), which is caused by defects in the function of the low-density lipoprotein (LDL) receptor pathway, occurs in about 1 in 350 people.
Treatment of hyperlipidemia began with disappointing results with niacin and bile acid sequestrants in the 1970s. A new class of low-density lipoprotein cholesterol (LDL-C) lowering medications called statins was introduced in 1990. While there was a great success with this new lipid-lowering therapy, researchers searched for genetic causes and new treatment options. Mutations were identified including the LDL receptors, apolipoprotein B, and proprotein convertase subtilisin/kexin type 9 (PCSK9). In 2015 the FDA approved both Praluent (alirocumab) and Repatha (evolocumab) for high-risk patients with familial hypercholesterolemia (FH) and atherosclerotic cardiovascular disease. Bococizumab was a PCSK9 inhibitor that Pfizer had in development that was withdrawn from studies in November 2016.
The list below includes the best PCSK9 inhibitors approved by the U.S. Food and Drug Administration (FDA) and their pricing:
List of PCSK9 inhibitors
Praluent is indicated to reduce the risk of cardiovascular disease complications and to lower LDL-C levels in patients with familial hypercholesterolemia.
Repatha is indicated to reduce the risk of cardiovascular disease complications and to lower LDL-C levels in patients with familial hypercholesterolemia.
Leqvio is indicated to lower LDL-C levels in patients with familial hypercholesterolemia or atherosclerotic cardiovascular disease.
What are PCSK9 inhibitors?
Familial hypercholesterolemia (FH) may be caused by mutations in the PCSK9 enzyme, which changes how well your body regulates your cholesterol levels. It is characterized by high total and LDL cholesterol levels, vascular disease, and cholesterol deposits in the tendons (xanthomas).
PCSK9 inhibitors are cholesterol-lowering drugs. They are used to treat FH and patients with a high cardiovascular risk that did not achieve sufficient LDL-cholesterol reductions with statin therapy. PCSK9 inhibitors can also decrease triglycerides and increase HDL-cholesterol levels.
Praluent (alirocumab) was studied in the ODYSSEY OUTCOMES trial that was published in the Journal of the American College of Cardiology (ACC). It showed significant reductions in cardiovascular events (heart attack, stroke, death) in patients with acute coronary syndrome.
Repatha (evolocumab) was shown to be effective at reducing the incidence of cardiovascular events compared to placebo in the FOURIER trial. Repatha therapy did not have any effect on mortality in this clinical trial.
How do PCSK9 inhibitors work?
Your liver makes a protein, PCSK9, that breaks down the LDL receptors that remove LDL cholesterol from your blood. The fewer LDL receptors you have, the higher your LDL cholesterol levels will be. PCSK9 inhibitors are monoclonal antibodies that block the action of the PCSK9 protein. PCSK9 inhibition results in more available LDL receptors and lower LDL cholesterol levels. Studies have shown that PCSK9 inhibitors reduce LDL cholesterol levels by an average of 47% which leads to a reduced risk of a heart attack by 27%.
PCSK9 inhibitors target hypercholesterolemia in a different way than statins, so they can be used together.
What conditions are PCSK9 inhibitors used to treat?
PCSK9 inhibitors are indicated:
- To reduce the risk of myocardial infarction (heart attack), ischemic stroke, and angina requiring hospitalization in patients with heart disease.
- Along with diet to treat patients with hyperlipidemia, including heterozygous familial hypercholesterolemia, to lower LDL-C levels. They may be used alone or in combination with other LDL-C lowering therapies.
- Along with diet and other LDL-C lowering therapies in patients with homozygous familial hypercholesterolemia to lower LDL-C levels.
PCSK9 inhibitors can be prescribed for both primary prevention (reducing the risk of cardiovascular events) and secondary prevention (preventing a second cardiovascular event). They are typically used if you have a statin intolerance or if other medications such as statins or Zetia (ezetimibe) don’t lower your blood cholesterol levels enough.
Are PCSK9 inhibitors safe?
When taken as prescribed, PCSK9 inhibitors are safe and effective. Make sure your healthcare provider is aware if you have any of the following:
- Hypersensitivity to a PCSK9 inhibitor
- Type 2 diabetes
You should also make sure they are aware of all the medications you take, including prescription drugs, over-the-counter medications, and supplements.
Can you take PCSK9 inhibitors while you are pregnant or breastfeeding?
There are no adequate studies regarding the use of PCSK9 inhibitors during pregnancy. There is a pregnancy safety study for Repatha (evolocumab) if it is used while pregnant. You should report exposure by contacting Amgen at 1-800-772-6436 or https://wwwext.amgen.com/products/global-patient-safety/adverse-event-reporting
There is also limited data on the use of PCSK9 inhibitors while breastfeeding. You should discuss the need for any medication with your doctor if you are pregnant, plan on becoming pregnant, or are breastfeeding.
What are the common side effects of PCSK9 inhibitors?
Some common adverse effects of PCSK9 inhibitors include:
- Injection site reactions
- Muscle pain
- Common cold symptoms
PCSK9 inhibitors can sometimes cause severe adverse events, including:
- Kidney impairment
- Liver impairment
This is not a complete list of side effects and we encourage you to consult with your healthcare professional for medical advice about any possible side effects.
How much do PCSK9 inhibitors cost?
PCSK9 inhibitors are very expensive with an average cost of around $20,000 per year.
You can purchase PCSK9 inhibitors for $49 per month from NiceRx if eligible for assistance. Prices at the pharmacy vary by location, strength, and quantity, as well as your insurance status.
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