FDA Approves Juxtapid for Rare Cholesterol Disorder

Orphan drug, capsule taken once a day, approved for homozygous familial hypercholesterolemia

THURSDAY, Dec. 27 (HealthDay News) -- The U.S. Food and Drug Administration has approved the orphan drug Juxtapid (lomitapide) for patients with homozygous familial hypercholesterolemia (HoFH), for use in combination with a low-fat diet and other lipid lowering treatments, according to a Dec. 26 press release.

Juxtapid, an orphan drug marketed by Cambridge, Mass.-based Aegerion Pharmaceuticals Inc., was evaluated in a trial including 29 patients with HoFH, an inherited disorder of high low-density lipoprotein (LDL) cholesterol. For those who tolerated the drug, levels of LDL cholesterol decreased by approximately one-half during the first 26 weeks.

Juxtapid, a capsule taken once a day, without food, at least two hours after the evening meal, carries a boxed warning regarding a serious risk of liver toxicity. Juxtapid also decreases the absorption of fat-soluble nutrients and, consequently, patients should take supplements that contain fat-soluble nutrients and essential fatty acids while taking Juxtapid. Juxtapid also interacts with several other medications.

"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," Eric Colman, M.D., deputy director of the Division of Metabolism and Endocrinology Products in the FDA's Center for Drug Evaluation and Research, said in a statement.

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