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ARTICLE | Company News

Aegerion gains on lomitapide briefing documents

October 16, 2012 1:33 AM UTC

Shares of Aegerion Pharmaceuticals Inc. (NASDAQ:AEGR) gained on Monday after FDA reviewers said lomitapide was "effective" at reducing LDL-C, total cholesterol, apolipoprotein B and triglycerides in patients with homozygous familial hypercholesterolemia (hoFH) in the product's only Phase III trial. The reviewers also said the company's proposed REMS would "support appropriate use of lomitapide, allowing it to be approved" for hoFH, while limiting its use in the larger hypercholesterolemia patient population. The comments came in briefing documents released ahead of Wednesday's Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) meeting to discuss an NDA for lomitapide. The small molecule microsomal triglyceride transfer protein (MTP) inhibitor has a Dec. 29 PDUFA date. Aegerion gained $1.50, or 9%, to $17.46 on Monday.

The committee will discuss whether lomitapide's 40.1% mean reduction in LDL-C at week 26 is expected to correlate with a reduction in cardiovascular morbidity and mortality in hoFH patients, given that the product's effect on cardiovascular outcomes will not be determined due to the disease's rarity. Reviewers did express concern about the effects of lomitapide on liver fat accumulation, liver enzymes, and on the absorption of fat-soluble vitamins and essential fatty acids. According to the documents, 11 of 29 patients in the Phase III trial had elevations in alanine aminotransferase (ALT) levels of at least three times over the upper limit of normal. However, the elevations were reversible, with improvements within 2-4 weeks of dose modification. ...