FDA Approves Amgen's Repatha to Lower LDL Cholesterol

August 27, 2015—Amgen announced that the US Food and Drug Administration (FDA) has approved the company’s new cholesterol-lowering medication, the Repatha (evolocumab) injection. Repatha is a human monoclonal antibody that inhibits proprotein convertase subtilisin/kexin type 9 (PCSK9), a protein that reduces the liver's ability to remove low-density lipoprotein cholesterol (LDL-C) from the blood. On June 10, the FDA announced that its Endocrinologic and Metabolic Drugs Advisory Committee voted to recommend approval of Repatha for the treatment of high cholesterol.

Repatha is expected to be available in the United States next week. Marketing authorization for Repatha in Europe was announced on July 21, 2015.

According to the company, Repatha is indicated as an adjunct to diet and maximally tolerated statin therapy for the treatment of adults with heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease (ASCVD), who require additional lowering of LDL-C, as well as an adjunct to diet and other LDL-lowering therapies for the treatment of patients with homozygous familial hypercholesterolemia (HoFH), who require additional lowering of LDL-C. The effect of Repatha on cardiovascular morbidity and mortality has not been determined. 

Marc Sabatine, MD, who is Chairman of the TIMI Study Group, commented in Amgen’s press release, “Through PCSK9 inhibition, evolocumab substantially reduces LDL or ‘bad’ cholesterol, a well-validated, modifiable risk factor for cardiovascular disease. Many patients still require further LDL cholesterol lowering and evolocumab now offers an important new treatment option for them.” Dr. Sabatine is the Lewis Dexter, MD, Distinguished Chair in Cardiovascular Medicine at Brigham and Women’s Hospital, and Professor of Medicine, Harvard Medical School in Boston, Massachusetts.

The company advised that in phase-3 trials, adding Repatha to background lipid-lowering therapy that included statins resulted in intensive reductions in LDL-C levels with favorable effects on other lipid parameters. In patients with clinical ASCVD or HeFH, Repatha reduced LDL-C by approximately 54% to 77% compared with placebo. In a pivotal phase-3 trial, 90% of clinical ASCVD patients who received Repatha in addition to maximum doses of statins achieved a LDL-C level < 70 mg/dL. In patients with HoFH, Repatha reduced LDL-C by approximately 30% compared with placebo.

The company advised that the GLAGOV intravascular ultrasound study is underway to determine the effect of Repatha on coronary atherosclerosis in approximately 950 patients undergoing cardiac catheterization to test the hypothesis of robust LDL-C reduction leading to a reduction or a change in the buildup of plaque in the arteries. Results from the GLAGOV study are expected in 2016.

Additionally, the FOURIER outcomes trial is designed to evaluate whether treatment with Repatha in combination with statin therapy compared to placebo plus statin therapy reduces the risk of recurrent cardiovascular events in patients with high cholesterol and clinically evident cardiovascular disease and completed patient enrollment in June 2015. Results from the approximately 27,500-patient, event-driven FOURIER study are expected no later than 2017.