Lomitapide Approved for Homozygous Familial Hypercholesterolemia

FDA Approves Lomitapide for Homozygous FH With Boxed Warning

December 24, 2012

The FDA has approved lomitapide (Aegerion Pharmaceuticals, Cambridge, MA) as an adjunct to a low-fat diet and other lipid-lowering drugs, with or without LDL apheresis, to reduce LDL-C, total cholesterol (TC), apolipoprotein B (apoB), and non-HDL-C in patients with homozygous familial hypercholesterolemia (FH) [1]. It will be known by the brand name Juxtapid.

The clearance follows the recommendation for approval by the FDA's Endocrinologic and Metabolic Drugs Advisory Committee in October by a vote of 13 to 2.

Back then, some panel members were concerned about the potential for liver toxicity with lomitapide, but others felt the risk-to-benefit profile was favorable, given the high risk associated with the extreme LDL-C levels in patients with homozygous FH. Because of this concern, the product will carry a boxed warning stating the risk of hepatotoxicity and the company has made a commitment to the FDA to conduct a post-approval, observational cohort study.

Lomitapide will be available only through a restricted program called the Juxtapid Risk Evaluation and Mitigation Strategy (REMS). Aegerion will certify all healthcare providers who prescribe Juxtapid and all pharmacies that dispense the medicine. The goal of the REMS is to educate prescribers about the risk of hepatotoxicity associated with the use of Juxtapid and the need to monitor patients during treatment as per product labeling, and to ensure that access to therapy is restricted to patients with a clinical or laboratory diagnosis consistent with homozygous FH.

The company notes that the safety and effectiveness of lomitapide has not been established in pediatric patients or those with hypercholesterolemia who do not have homozygous FH, nor has its effect on cardiovascular morbidity and mortality been determined.

As part of the planned launch of the product, the company has developed a comprehensive support services program for patients and their healthcare providers. For more information, call 1-85Juxtapid (1-855-898-2743).

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