Daiichi Sankyo Europe, Esperion Announce Validation of MAA for Bempedoic Acid and the Bempedoic Acid/Ezetimibe Fixed Dose Combination Tablet

Daiichi Sankyo Europe and Esperion Therapeutics announced the validation of the Marketing Authorisation Application (MAA) for bempedoic acid and the bempedoic acid / ezetimibe fixed dose combination tablet by the European Medicines Agency (EMA). Validation confirms that the application is complete and commences the scientific review process by the EMA's Committee for Medicinal Products for Human Use (CHMP).

Bempedoic acid is an oral, once-daily ATP Citrate Lyase (ACL) inhibitor that reduces cholesterol and fatty acid synthesis in the liver. Bempedoic acid and the bempedoic acid / ezetimibe fixed dose combination tablet could become an important treatment option for patients with hypercholesterolemia who are not yet at their target LDL-C goals with existing therapies or who have statin intolerance.

This MAA validation for the LDL-C lowering indication is based on the successful completion of the global pivotal Phase 3 program, which demonstrated bempedoic acid was well tolerated and efficacious for chronic use in almost 4,800 patients. Patients treated with bempedoic acid produced an additional 20 percent LDL-C lowering when used with maximally tolerated statins, up to 30 percent LDL-C lowering as monotherapy, 35 percent LDL-C lowering in combination with ezetimibe when used with maximally tolerated statins and up to 48 percent LDL-C lowering in combination with ezetimibe as monotherapy. In the Phase 3 trials, rates of treatment-emergent adverse events, muscle-related adverse events and discontinuations were similar in the bempedoic acid and placebo treatment group.

There is a significant unmet need for patients with elevated LDL-C who are not reaching their treatment goals with existing therapies. This is particularly true for patients taking statins only at the maximum tolerated dose or are statin intolerant and need additional LDL-C lowering. Bempedoic acid has a liver specific mode of action and therefore has the potential to avoid the muscle related adverse drug reactions (ADRs) associated with statin therapy. Bempedoic acid can be used in combination with other lipid lowering drugs and will offer a once-daily, convienent, oral option for patients not at target goals.

"Unlike some of the historical cardiovascular players who have decided to focus their effort on other therapeutic areas, we remain committed to the cardiovascular field. With bempedoic acid, our first-in-class treatment, we build on our dedication to address critical unmet needs for patients in Europe who have limited options and who are not reaching their target LDL-C level," said Benoit Creveau, Head of Marketing Cardiovascular at Daiichi Sankyo Europe.

"We are very pleased to partner with Daiichi Sankyo Europe to establish bempedoic acid as the most preferred LDL-C lowering treatment option after statins for patients and physicians in Europe. We expect Daiichi Sankyo Europe's significant cardiovascular reach and expertise will help establish bempedoic acid and the bempedoic acid / ezetimibe fixed dose combination tablet as important tools in physicians' and patients' fight against elevated LDL-C levels," said Tim Mayleben, President and Chief Executive Officer of Esperion.

"The results from the pivotal Phase 3 LDL-C development program of bempedoic acid and bempedoic acid / ezetimibe fixed dose combination tablet provide compelling evidence that bempedoic acid is an efficacious and well tolerated therapeutic option and we hope to make it available as soon as possible to European patients," adds Rodney Smith, MD, Head of Medical Affairs at Daiichi Sankyo Europe.

Esperion completed its Phase 3 LDL-C development program of bempedoic acid and the bempedoic acid / ezetimibe fixed dose combination tablet in October 2018. This robust development program forms the bases for FDA and EMA approval decisions that are expected during the first half of 2020. The global cardiovascular outcomes trial of bempedoic acid, CLEAR Outcomes, is ongoing, and cardiovascular risk reduction data are expected during 2022.

Through the complementary mechanisms of action of inhibition of cholesterol synthesis (bempedoic acid) and inhibition of cholesterol absorption (ezetimibe), the bempedoic acid / ezetimibe fixed dose combination tablet is a non-statin, orally available, once-daily, LDL-C lowering therapy. Inhibition of ATP Citrate Lyase (ACL) by bempedoic acid reduces cholesterol biosynthesis and lowers LDL-C by up-regulating the LDL receptor. Inhibition of Niemann-Pick C1-Like 1 (NPC1L1) by ezetimibe results in reduced absorption of cholesterol from the gastrointestinal tract, thereby reducing delivery of cholesterol to the liver, which in turn upregulates the LDL receptors. Phase 3 data demonstrated that this well tolerated combination results in a 35 percent lowering of LDL-C when used with maximally tolerated statins, a 43 percent lowering of LDL-C when used as a monotherapy, and a 34 percent reduction in high sensitivity C-reactive protein (hsCRP). Rates of treatment-emergent adverse events, muscle-related adverse events and discontinuations were similar in the bempedoic acid and placebo treatment groups.

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