FDA approves lomitapide for hoFH

FDA approved Juxtapid lomitapide from Aegerion Pharmaceuticals Inc. (NASDAQ:AEGR) on Monday to treat patients with homozygous familial hypercholesterolemia (hoFH). The small molecule microsomal triglyceride transfer protein (MTP) inhibitor is approved as an adjunct to a low-fat diet and other lipid-lowering therapies, including LDL apheresis where available, to reduce LDL-C, total cholesterol, apolipoprotein B (APOB) and non-HDL-C in hoFH patients. Aegerion said it plans to launch Juxtapid in January at an annual price of $200,000-$300,000 per patient.

Juxtapid was approved with a REMS under which Aegerion will certify all prescribers and pharmacies in an effort to educate prescribers about the risk of hepatotoxicity and the need to monitor patients during treatment. The REMS also now requires patients to have a "clinical or laboratory diagnosis consistent with hoFH," but does not specify how the diagnosis must be made. Previously, the REMS did not require a specific diagnosis to dispense the drug. Aegerion said it estimates that based on a genetic test there are about 300 hoFH patients in the U.S. However, the company said the number of hoFH patients could be much larger if a diagnosis is based on other clinical factors, including high cholesterol. On a conference call to discuss the approval, Aegerion said the "total adjustable market" for Juxtapid could be as many as 3,000 patients in the U.S. ...