Otsuka Pharmaceutical Co., Ltd. (Otsuka) announces the submission of a New Drug Application (NDA) to the Japanese Ministry of Health, Labour and Welfare for the manufacture and sale in Japan of bempedoic acid for the treatment of hypercholesterolemia and familial hypercholesterolemia.

Bempedoic acid, created by Esperion Therapeutics, Inc. (Esperion), has a novel mechanism of action that inhibits a cholesterol synthesis pathway by acting on ATP (adenosine triphosphate) citrate lyase, a citrate-degrading enzyme in the liver. Bempedoic acid is marketed for the treatment of hypercholesterolemia in several regions around the world, including the United States and Europe. In 2020, Otsuka acquired exclusive development and commercialization rights for bempedoic acid in Japan from Esperion and is currently developing it domestically.

(https://www.otsuka.co.jp/en/company/newsreleases/2020/20200420_1.html)

This Phase 3 trial (NCT05683340) was conducted as a placebo-controlled, randomized, multicenter, double-blind, parallel-group comparative study, in 96 patients with high LDL cholesterol and in whom statins have insufficient effect or cannot be tolerated. Trial participants were administered either 180 mg of bempedoic acid or a placebo, orally, once a day, for 12 weeks to evaluate the efficacy and safety of bempedoic acid. The percentage change from baseline in LDL-C at Week 12, the primary endpoint, was -25.25 percent in the group receiving bempedoic acid group and -3.46 percent in the placebo group, demonstrating positive outcomes with statistical significance compared to placebo (p<0.001). Furthermore, the safety and tolerability of bempedoic acid were consistent with findings from previous trials, and no serious adverse events were observed.

Some patients with hypercholesterolemia are unable to achieve their target values even when taking statins (insufficient response to statins), or they are unable to continue taking statins due to the occurrence of adverse events associated with statin use (statin intolerance). This drug candidate in Japan is expected to become a new treatment option for hypercholesterolemic patients with insufficient response to statins or statin intolerance.

Otsuka Pharmaceutical will continue to deliver innovative products to meet unmet medical needs around the world.