ANN ARBOR, Mich.—Esperion Therapeutics Inc., a late-stage pharmaceutical company focused on developing and commercializing first-in-class oral therapies for the treatment of patients with elevated low-density lipoprotein cholesterol (LDL-C), recently provided updates on the bempedoic acid (ETC-1002) clinical development program and regulatory plans. The clinical program has two major components: the global pivotal Phase 3 efficacy and safety studies, and the planned global cardiovascular outcomes trial (CVOT) in patients with elevated LDL-C levels who are unable to tolerate statins (statin intolerance).
The company intends to initiate global pivotal Phase 3 efficacy studies for LDL-C lowering in patients with hypercholesterolemia, and a CVOT specifically in statin-intolerant patients who are at high risk for cardiovascular (CV) disease, in the fourth quarter of 2016.
For Europe, the company engaged with the European Medicines Agency, resulting in alignment on the definition of statin intolerance and key design features of the global Phase 3 LDL-C lowering program and CVOT. The planned LDL-C lowering efficacy studies, together with the ongoing long-term safety and tolerability study (1002-040) initiated in January 2016, are designed to support a submission for an LDL-C lowering Marketing Authorization Application for bempedoic acid in patients with elevated LDL-C, including statin-intolerant patients, in Europe by 2019.
For the United States, the company engaged with the U.S. Food and Drug Administration, resulting in alignment on the definition of statin intolerance, key design features of the CVOT, and the regulatory approval pathway for a CV disease risk reduction indication in statin-intolerant patients for bempedoic acid. However, the FDA did not provide clarity on a regulatory pathway for an LDL-C lowering indication in the United States in statin-intolerant patients at this time. The Agency indicated its position regarding an LDL-C lowering indication could be impacted by potential future changes in their view of LDL-C lowering as a surrogate endpoint or the possibility of a shift in the future standard-of-care for statin-intolerant patients with elevated LDL-C levels.