Juxtapid earns FDA approval in homozygous familial hypercholesterolemia

The FDA has approved lomitapide to be used in combination with a low-fat diet and other lipid-lowering treatments for the reduction of LDL, non-HDL and total cholesterol, and apolipoprotein B in patients with homozygous familial hypercholesterolemia, according to a press release.

The agency based its decision on a clinical trial that evaluated safety and efficacy of lomitapide (Juxtapid, Aegerion Pharmaceuticals) in 29 patients with homozygous familial hypercholesterolemia. LDL cholesterol decreased by approximately 50% during the first 26 weeks of treatment among patients who tolerated the drug. Common adverse events included diarrhea, nausea, vomiting, indigestion and abdominal pain.

Lomitapide also is associated with liver enzyme abnormalities and accumulation of fat in the liver and therefore carries a boxed warning regarding serious risk for liver toxicity. Additionally, the drug reduces the absorption of fat-soluble nutrients and interacts with several other medications.

The FDA approved lomitapide with a Risk Evaluation and Mitigation Strategy that requires prescriber and pharmacy certification and documentation of safe-use conditions, such as a prescription authorization form that will be required for each new prescription, according to the release.

Along with approval, the agency also is requiring three post-marketing studies, including an animal study to evaluate the potential for toxicity in children and adolescents; a long-term registry of patients with homozygous familial hypercholesterolemia treated with lomitapide to assess long-term safety; and an enhanced pharmacovigilance program to monitor reports of malignancy, teratogenicity and hepatic abnormalities.

In October, the FDA Endocrinologic and Metabolic Drugs Advisory Committee recommended approval of lomitapide in a 13-2 vote despite concerns about the drug’s hepatic safety.