Amgen submits application to FDA seeking approval of Repatha injection

Approved by the FDA on Aug. 27, 2015, Repatha is a human monoclonal antibody that inhibits proprotein convertase subtilisin/kexin type 9 (PCSK9). PCSK9 is a protein that reduces the liver’s ability to remove low-density lipoprotein cholesterol (LDL-C), or "bad" cholesterol, from the blood.1

Repatha is approved as an adjunct to diet and maximally tolerated statins in patients with heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic cardiovascular disease (ASCVD), who require additional lowering of LDL-C; and 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.

Amgen executive vice president of research and development Sean Harper said: "We are very excited about the recent approval of Repatha in the U.S. as a new treatment option for patients who are in need of lowering their LDL cholesterol.

"In addition to the SureClick® autoinjector, we are developing this monthly single injection to provide patients with another option to administer Repatha every month. Patients who are in need of lowering their cholesterol levels are often on more than one medication and some may prefer a single-dose option for receiving Repatha once monthly."

Repatha is available as a single-use 140 mg/mL prefilled SureClick autoinjector or prefilled syringe that patients can self-administer at the recommended dose for adults of 140 mg every two weeks or 420 mg once a month. For patients with HoFH, the recommended dose is 420 mg once a month.

About High Cholesterol

Elevated low-density lipoprotein cholesterol (LDL-C) is an abnormality of cholesterol and/or fats in the blood, and is recognized as a major risk factor for cardiovascular disease.2,3 In the U.S., there are approximately 11 million people with atherosclerotic cardiovascular disease (ASCVD) and/or familial hypercholesterolemia (FH), who have uncontrolled levels of LDL-C over 70 mg/dL, despite treatment with statins or other cholesterol-lowering therapies.4,5 Familial hypercholesterolemia is caused by genetic mutations that lead to high levels of LDL-C at an early age.6 It is estimated that one million people in the U.S. have FH (heterozygous and homozygous forms), yet less than one percent are diagnosed.7

About RepathaTM (evolocumab)

RepathaTM (evolocumab) is a human monoclonal antibody that inhibits proprotein convertase subtilisin/kexin type 9 (PCSK9).1 Repatha binds to PCSK9 and inhibits circulating PCSK9 from binding to the low-density lipoprotein (LDL) receptor (LDLR), preventing PCSK9-mediated LDLR degradation and permitting LDLR to recycle back to the liver cell surface. By inhibiting the binding of PCSK9 to LDLR, Repatha increases the number of LDLRs available to clear LDL from the blood, thereby lowering LDL-C levels.8

GLAGOV, the 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 build-up of plaque in the arteries. Results from the GLAGOV study are expected in 2016.

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 FOURIER study are expected no later than 2017 (event-driven).