HoFH is a rare, life-threatening condition that occurs in approximately 1 in 250,000 individuals. Patients with HoFH have two mutations in a small group of genes that controls the way the body clears cholesterol. As a result, patients have extremely high circulating levels of low-density lipoprotein cholesterol (LDL-C), commonly known as "bad cholesterol." These cholesterol levels can range as high as 500-1,000 mg/dL, more than four times the normal levels.
Patients with HoFH can develop premature cardiovascular disease, including heart attack and heart disease, when they are teenagers or in their 20s, and many do not improve substantially with other cholesterol-lowering drugs. Without aggressive treatment, patients may die before age 30.
Praluent works by stopping the proprotein convertase subtilisin kexin type 9 (PCSK9) protein. PCSK9 controls the number of LDL receptors, which are proteins that help remove LDL-C from the bloodstream and enable the breakdown of fats and cholesterol. By blocking PCSK9, Praluent allows faster removal of LDL-C from the blood to lower cholesterol levels. Praluent is given by needle injection under the skin every two weeks.
The effectiveness and safety of Praluent were evaluated in a 12-week, double-blind, randomized trial among adult patients with HoFH. In the trial, 45 patients received 150 mg of Praluent every two weeks and 24 patients received a placebo. Patients were taking other therapies to lower LDL-C as well.
The primary measure of effectiveness was the percent change in LDL-C from the beginning of treatment to week 12. At week 12, patients receiving Praluent had an average 27% decrease in LDL-C whereas patients on the placebo had an average 9% increase.
Common side effects of Praluent are nasopharyngitis (cold), injection site reactions, and influenza. Serious hypersensitivity (allergic) reactions have occurred among people taking Praluent.
Praluent was originally approved in 2015. It is also approved to reduce the risk of heart attack, stroke and unstable angina (a condition in which the heart does not receive enough blood flow and oxygen) requiring hospitalization in adults with cardiovascular disease. In addition, Praluent is approved to be used alone or in addition to diet or other therapies to treat primary hyperlipidemia (certain forms of high cholesterol).
FDA granted approval of Praluent to Regeneron Pharmaceuticals, Inc.
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