Less than a month after landing a positive opinion from the EMA's CHMP, Esperion Therapeutics Inc. has won FDA approval for bempedoic acid as an adjunct to diet and maximally tolerated statin therapy. The approval covers the treatment of adults with heterozygous familial hypercholesterolemia (HeFH) or established atherosclerotic cardiovascular disease who require additional lowering of low-density lipoprotein cholesterol (LDL-C). It will be branded as Nexletol, in a variation from the expected European brand name, Nilemdo. An EMA review of the company's MAA is ongoing. The price will be about $10 a day, a representative told BioWorld.

"Even with maximally tolerated statins, which may mean no statin at all, some of these patients can’t achieve their LDL-C goals," said Tim Mayleben, Esperion's president and CEO. "Today’s approval provides them with a new medicine to go along with a healthy diet."

Shares (NASDAQ:ESPR) of the Ann Arbor, Mich.-based company fell by nearly 15% on announcement of the approval, though recovered some ground as investors digested the news.

Nexletol, a prodrug, is a first-in-class oral ATP citrate lyase inhibitor that lowers LDL-C by reducing cholesterol biosynthesis and up-regulating the LDL receptor. In phase III studies conducted in more than 4,000 patients, it demonstrated up to 18% placebo-corrected LDL-C lowering when used with moderate- and high-intensity statins and 21% to 28% placebo-corrected LDL-C lowering when used with low-dose or no background statin.

Upon launch, the new medicine will face competition from two key drugs in the cholesterol market, the PCSK9 inhibitors Repatha (evolocumab) from Amgen Inc. and Praluent (alirocumab) from Sanofi SA. Though Nexletol will have an advantage on dosing convenience, PCSK9 inhibitors dramatically lower LDL-C levels, by up to 60%, when combined with a statin, notes a Cortellis analysis, meaning that such drugs are likely to be preferred in patients with the hardest to treat cholesterol levels, such as those with HeFH or with heart disease.

An FDA review of a tablet combining bempedoic acid and ezetimibe, a cholesterol absorption inhibitor first commercialized by Merck & Co. Inc. but now generic, is set for Feb. 26.

The effect of bempedoic acid on cardiovascular morbidity and mortality has not yet been determined. But Esperion has initiated a global cardiovascular outcomes trial to assess its effect on the occurrence of major cardiovascular events in patients with, or at high risk for, cardiovascular disease.

Esperion submitted its MAAs for bempedoic acid and the bempedoic acid/ezetimibe combination tablet to the EMA on Feb. 11, 2019. Shortly before that, the company inked a license and collaboration agreement with Daiichi Sankyo Europe GmbH, granting it exclusive commercialization rights to both the monotherapy and combination products in the European Economic Area and Switzerland.

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