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New Drugs Approved for People with Hard to Treat High Cholesterol

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September 24, 2015

The U.S. Food and Drug Administration (FDA) recently approved the first drugs in a new class of cholesterol-lowering medications for certain people whose cholesterol levels have not responded adequately to treatment with statins. The specific approvals are for people who have heart disease or a history of heart attacks or strokes, or have hereditary high cholesterol.

High cholesterol, specifically an increased level of low-density lipoprotein cholesterol (LDL), is linked to an increased risk of cardiovascular disease (CVD). Excess cholesterol in the blood may be deposited in plaques on blood vessel walls, leading to hardening of the arteries (atherosclerosis) and increased risk of heart attacks and stroke. Blood tests are used to check cholesterol levels and help determine CVD risk.

In the U.S., statins have been used for decades to treat high LDL and lower CVD risk. Comprehensive guidelines from the American Heart Association and the American College of Cardiology, supported by data from two recent studies, recommend that Americans with heart risk above a certain threshold be treated with statins. Though often prescribed to lower cholesterol, in some people statins are either not effective or not effective enough. These people may be helped by the new class of drugs approved by the FDA.

The drugs are antibodies that target a specific protein called proprotein convertase subtilisin kexin type 9 (PCSK9) and are called PCSK9 inhibitors. The protein PCSK9 reduces the number of receptors on the liver that remove LDL from the blood. By blocking PCSK9, the drugs allow more receptors to be available to clear LDL from the blood. So far, the FDA has approved Praluent and Repatha, and at least one more PCSK9 inhibitor is being reviewed by the FDA.

Both medications were tested in multiple clinical trials against placebos. The trials included people with high cholesterol who already follow healthy diets and take the highest dose of statins they can tolerate. The new drugs were able to significantly further reduce LDL cholesterol levels.

Since these drugs are antibodies, they are currently only available in injectable form. They come as pre-filled auto-injectors that patients administer themselves every two weeks or once a month. Though shown to be effective, there are some drawbacks to both drugs. Common side effects include itching, swelling, pain or bruising at the injection site, and back pain, colds, flu and some allergic reactions requiring hospitalization. The drugs are expected to cost about $14,000 per year and insurers are currently considering whether to cover them.

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NOTE: This article is based on research that utilizes the sources cited here as well as the collective experience of the Lab Tests Online Editorial Review Board. This article is periodically reviewed by the Editorial Board and may be updated as a result of the review. Any new sources cited will be added to the list and distinguished from the original sources used. To access online sources, copy and paste the URL into your browser.

(August 27, 2015) FDA approves Repatha to treat certain patients with high cholesterol. Available online at http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm460082.htm. Accessed September 9, 2015.

(July 24, 2015) FDA Approves Praluent to treat certain patients with High cholesterol. Available online at http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm455883.htm. Accessed September 9, 2015.

(August 27, 2015) FDA Approves Amgen's New Cholesterol-Lowering Medication Repatha™ (evolocumab). Available online at https://www.amgen.com/media/media_pr_detail.jsp?year=2015&releaseID=208283. Accessed September 9, 2015.

(July 24, 2015) Sanofi and Regeneron Announce FDA Approval of Praluent (alirocumab) Injection, the first PCSK9 Inhibitor in the U.S. for the Treatment of High LDL Cholesterol in Adult Patients. Available online at http://en.sanofi.com/NasdaQ_OMX/local/press_releases/sanofi_and_regeneron_announce__1941221_24-07-2015!21_24_28.aspx. Accessed September 9, 2015.

(September 8, 2015) ICER Draft Report on Effectiveness, Value, and Pricing Benchmarks for PCSK9 Inhibitors for High Cholesterol Posted for Public Comment. Available online at http://www.icer-review.org/pcsk9-draft-report-release/. Accessed September 9, 2015.