The FDA on Friday approved Esperion Therapeutics' Nexletol (bempedoic acid) for the treatment of adults with heterozygous familial hypercholesterolaemia or established atherosclerotic cardiovascular disease who require additional lowering of LDL cholesterol. The once-daily oral drug, which will be available as of March 30, will be priced at around $10 per tablet, the company said.
CEO Tim Mayleben pointed out that not all patients can tolerate statins, and "some of these patients can't achieve their LDL-cholesterol goals," adding that "today's approval provides them with a new medicine to go along with a healthy diet." According to Esperion, by inhibiting the ACL enzyme, Nexletol reduces cholesterol synthesis, resulting in LDL receptor upregulation and increased clearance of LDL from the bloodstream. The company said it is targeting about 9 million patients who are not achieving adequate results with statins, as well as about 9 million others who are considered unsuitable for statin use.
The FDA filing for Nexletol is based on data from Phase III studies  conducted in more than 4000 patients demonstrating up to 18% placebo-corrected LDL-cholesterol lowering when used with moderate- and high-intensity statins, as well as 21% to 28% placebo-corrected LDL-cholesterol lowering when used with low-dose or no background statins. In addition, Phase III data for Nexletol plus ezetimibe, which is also under FDA review with a decision expected on February 26, showed that the once-daily combination drug led to a 29% lowering of LDL-cholesterol when used with maximally tolerated statins, and a 44% reduction when used with no background statin.
Stifel analyst Derek Archila expects the drug to bring in sales of about $3 billion by 2029 at its peak. However, shares in Esperion still fell as much as nearly 11% on Friday.
Needham analyst Chad Messer said that although the label for Nexletol is slightly more restrictive than expected, it is nevertheless in line with how the FDA treated the injectable PCSK9 inhibitors Repatha (evolocumab), sold by Amgen, and Praluent (alirocumab), which is jointly marketed by Sanofi and Regeneron Pharmaceuticals. "Overall, I don't think it's a bad label at all, but this stock has a history of overreacting to small issues," he said. The analyst also noted that the label warns against the potential for tendon rupture and some dosing restrictions when used in conjunction with other drugs, but Esperion said the label is in line with expectations.
Last month, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency issued a positive opinion  for Nexletol to treat hypercholesterolaemia and mixed dyslipidaemia.
For related analysis, see ViewPoints: Street warming to Esperion's bempedoic acid – for now?