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FDA Approves Evolocumab (Repatha) to Treat Certain Patients With High Cholesterol

PCSK9 inhibitor reduces LDL-C levels

The FDA has given the green light to evolocumab (Repatha, Amgen) injection for some patients who are unable to get their low-density lipoprotein cholesterol (LDL-C) under control with current treatment options.

Evolocumab, the second drug approved in a new class of agents known as proprotein convertase subtilisin/kexin type 9 (PCSK9) inhibitors, is indicated for use in addition to diet and maximally tolerated statin therapy in adult patients with heterozygous familial hypercholesterolemia (HeFH), homozygous familial hypercholesterolemia (HoFH), or clinical atherosclerotic cardiovascular disease, such as heart attacks or strokes, who require additional lowering of LDL-C.

Familial hypercholesterolemia (encompassing both HeFH and HoFH) is an inherited condition that causes high levels of LDL-C. A high concentration of LDL-C in the blood is linked to cardiovascular or heart 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, approximately 610,000 people die of heart disease in the U.S. every year –– that equals one in every four deaths.

Evolocumab is an antibody that targets the PCSK9 protein. PCSK9 reduces the number of receptors on the liver that remove LDL-C from the blood. By blocking the ability of PCSK9 to work, more receptors are available to get rid of LDL-C from the blood and, as a result, reduce LDL-C levels.

The efficacy and safety of evolocumab were evaluated in one 52-week placebo-controlled trial and in eight 12-week placebo-controlled trials in participants with primary hyperlipidemia, including two studies that specifically enrolled participants with HeFH and one that enrolled participants with HoFH. In one of the 12-week studies, 329 participants with HeFH, who required additional lowering of LDL-C despite statins with or without other lipid-lowering therapies, were randomly assigned to receive evolocumab or placebo for 12 weeks. The participants receiving evolocumab had an average reduction in LDL-C of approximately 60% compared with placebo.

The most common adverse events associated with evolocumab included nasopharyngitis, upper respiratory tract infection, flu, back pain, and injection-site reactions, such as redness, pain, or bruising. Allergic reactions, such as rash and hives, have been reported with the use of evolocumab. Patients should stop using evolocumab and get medical help if they experience symptoms of a serious allergic reaction.

Numerous clinical trials have demonstrated that statins lower the risk of having a heart attack or stroke. A study evaluating the effect of adding evolocumab to statins for reducing cardiovascular risk is ongoing.

Source: FDA; August 27, 2015.


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