Press Announcements > FDA approves new orphan drug for rare cholesterol disorder
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`FDA NEWS RELEASE
`For Immediate Release: Dec. 26, 2012
`Media Inquiries: Stephanie Yao, 301-796-0394, stephanie.yao@fda.hhs.gov
`Consumer Inquiries: 888-INFO-FDA
`FDA approves new orphan drug for rare cholesterol disorder
`On Dec. 21, the U.S. Food and Drug Administration approved Juxtapid (lomitapide) to reduce low-density
`lipoprotein (LDL) cholesterol, total cholesterol, apolipoprotein B, and non-high-density lipoprotein (non-HDL)
`cholesterol in patients with homozygous familial hypercholesterolemia (HoFH). Juxtapid is intended for use in
`combination with a low fat diet and other lipid-lowering treatments.
`HoFH is a rare inherited condition that makes the body unable to remove LDL cholesterol, often called the “bad”
`cholesterol, from the blood, causing abnormally high levels of circulating LDL cholesterol. In the United States,
`HoFH occurs in approximately one in one million individuals. For those with HoFH, heart attacks and death often
`occur before age 30. Juxtapid works by impairing the creation of the lipid particles that ultimately give rise to LDL.
`Juxtapid is a capsule taken once a day, without food, and at least two hours after the evening meal. Patients
`should take supplements that contain fat-soluble vitamins and essential fatty acids daily while taking Juxtapid.
`“Juxtapid, in addition to diet changes and other cholesterol-lowering treatments, is a new option for those
`suffering with HoFH and the serious health consequences resulting from this condition,” said Eric Colman, M.D.,
`deputy director of the Division of Metabolism and Endocrinology Products in the FDA’s Center for Drug Evaluation
`and Research.
`The safety and effectiveness of Juxtapid were evaluated in a clinical trial of 29 patients with HoFH. On average,
`levels of LDL cholesterol fell by approximately one-half during the first 26 weeks among those who tolerated the
`drug. Juxtapid carries a Boxed Warning regarding a serious risk of liver toxicity because it is associated with liver
`enzyme abnormalities and accumulation of fat in the liver, which could potentially lead to progressive liver disease
`with chronic use. Juxtapid also reduces the absorption of fat-soluble nutrients and interacts with several other
`medications.
`The FDA approved Juxtapid with a Risk Evaluation and Mitigation Strategy (REMS) that consists of elements to
`ensure safe use including prescriber and pharmacy certification and documentation of safe-use conditions
`consisting of a prescription authorization form that will be required to accompany each new prescription.
`The FDA is requiring three postmarketing studies for Juxtapid: an animal study to evaluate the potential for
`toxicity in children and teens; a long-term registry of patients with HoFH treated with Juxtapid to determine the
`long-term safety; and an enhanced pharmacovigilance program to monitor reports of malignancy, teratogenicity,
`and hepatic abnormalities.
`The most common adverse reactions in the clinical trial included diarrhea, nausea, vomiting, indigestion, and
`abdominal pain.
`Juxtapid is marketed by Cambridge, Mass.-based Aegerion Pharmaceuticals Inc.
`For more information:
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`http://www.fda.gov/NewsEvents/Newsroom/PressAnnouncements/ucm333285.htm
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`12/7/2015
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`Press Announcements > FDA approves new orphan drug for rare cholesterol disorder
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