On July 24, the Food and Drug Administration (FDA) announced that it had approved Praluent (alirocumab) injection for the treatment of elevated cholesterol levels for individuals who are unable to control their cholesterol with statins. Praluent is the first cholesterol-lowering treatment approved among a new class of drugs known as proprotein convertase subtilisin kexin type 9 (PCSK9) inhibitors. However, the drug, manufactured by Regeneron Pharmaceuticals Inc. and Sanofi SA, comes at a hefty price: $14,600 per year; thus, fueling the growing concern regarding drug costs.
The FDA approved Praluent is approved for use in addition to diet and maximally tolerated statin therapy in adult patients with heterozygous familial hypercholesterolemia (HeFH) or patients with clinical atherosclerotic cardiovascular disease such as myocardial infarctions (heart attacks) or strokes, who require additional lowering of low-density lipoprotein (LDL; “bad”) cholesterol.
The FDA explains that HeFH is an inherited condition that causes high levels of low-density lipoprotein (LDL) cholesterol. A high level of LDL cholesterol in the blood is associated with cardiovascular disease. Heart disease is the number one cause of death for Americans, both men and women. According to the Centers for Disease Control and Prevention (CDC), approximately 610,000 individuals die of heart disease in the US every year (one in every four deaths).
“Praluent provides another treatment option for patients with HeFH or with known cardiovascular disease who have not been able to lower their LDL cholesterol enough on statins,” explained John Jenkins, M.D., director of the Office of New Drugs, Center for Drug Evaluation and Research. He added, “The FDA strongly supports continued work to provide new and innovative options for the treatment and prevention of cardiovascular disease.”
Praluent is an antibody, which targets a specific protein, called PCSK9; the drug reduces the number of receptors on the liver that remove LDL cholesterol from the blood. By blocking PCSK9’s ability to function, more receptors are available to remove LDL cholesterol from the blood and, as a result, lower LDL cholesterol levels.
The effectiveness and safety of the medication were evaluated in five placebo-controlled trials, comprised of 2,476 individuals who received Praluent. All the participants had HeFH or were otherwise at high risk for heart attack or stroke; in addition, they were taking maximally tolerated doses of a statin, with or without other lipid‑lowering treatment. The participants taking Praluent were found to have an average reduction in LDL cholesterol ranging from 36-59%, compared to placebo.
A number of clinical trials have demonstrated that statins lower the risk of having a heart attack or stroke. Currently, a trial evaluating the effect of adding Praluent to statins on reducing cardiovascular risk is ongoing. The most common side-effects of Praluent include itching, swelling, pain, or bruising where injection is given, nasopharyngitis (inflammation of the nose and throat), and flu. Allergic reactions, such as hypersensitivity vasculitis (a skin rash usually appearing as purple-colored spots on the skin associated with inflammation of small blood vessels) and hypersensitivity reactions requiring hospitalization, have been reported with the use of Praluent. Patients should stop taking Praluent and seek medical attention if they experience symptoms of a serious allergic reaction.
Take home message: All medications have side-effects, sometimes serious, and many are expensive. Thus, changes in lifestyle should be tried first before taking them. Many individuals with elevated cholesterol levels are overweight; thus, these individuals should engage in a diet and exercise program to lower their body mass index (BMI) to normal levels. In many cases, it will result in a healthy cholesterol profile, if not, consult your healthcare provider for other options.