FDA approves first and only single monthly injection for a PCSK9 inhibitor

AmgenAmgen (NASDAQ:AMGN) today announced that the U.S. Food and Drug Administration (FDA) has approved the Repatha® (evolocumab) PushtronexTM system (on-body infusor with prefilled cartridge), a new, monthly single-dose administration option. (1) The Pushtronex system is a hands-free device designed to provide 420 mg of Repatha in a single dose. Repatha is a human monoclonal antibody that blocks a protein called proprotein convertase subtilisin/kexin type 9 (PCSK9), which inhibits the body's natural system for eliminating "bad" cholesterol (low-density lipoprotein cholesterol or LDL-C) from the blood.1 Repatha is the first and only PCSK9 inhibitor to offer a monthly single-dose delivery option.

"The Pushtronex system exemplifies Amgen's continued innovation and commitment to patients," said Sean E. Harper, M.D., executive vice president of Research and Development at Amgen. "Repatha is the only PCSK9 inhibitor with an approved monthly dose, and now the only one with a monthly single-dose administration. The FDA approval of the Pushtronex system offers another delivery option to patients who need the additional LDL cholesterol lowering that Repatha can provide."

In the U.S., 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; and as an adjunct to diet and other LDL-lowering therapies for the treatment of patients with homozygous familial hypercholesterolemia (HoFH) over age 13, who require additional lowering of LDL-C.(1) The effect of Repatha on cardiovascular morbidity and mortality has not been determined.

The new, single-use device was developed in collaboration with West Pharmaceutical Services, based on the SmartDose® technology platform, to provide patients with an additional dosing option for Repatha treatment. The device adheres to the body and patients are hands free during administration. Patients are able to perform moderate physical activities (such as walking, reaching or bending) as the 420 mg of Repatha is delivered subcutaneously.

The U.S. Wholesale Acquisition Cost (WAC) price of Repatha is $14,100 annually, whether it is delivered monthly via Pushtronex system or every two weeks via SureClick® autoinjector. Actual costs to patients, payers and health systems are anticipated to be lower as WAC pricing does not reflect discounts or rebates. Out-of-pocket costs to patients will vary depending on insurance status and eligibility for patient assistance. The Pushtronex system will be available to patients in the U.S. in early August.

Elevated LDL-C is an abnormality of cholesterol and/or fats in the blood and is recognized as a major risk factor for cardiovascular disease.(2-5) In the U.S., there are approximately 11 million people with 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.(6,7) FH is caused by genetic mutations that lead to high levels of LDL-C at an early age.(8) It is estimated that one million people in the U.S. have FH, yet less than one percent are diagnosed.(9)

About Repatha® (evolocumab)
Repatha® (evolocumab) is a human monoclonal antibody that inhibits proprotein convertase subtilisin/kexin type 9 (PCSK9). 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.(1)

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 the second half of 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 cardiovascular events in patients with high cholesterol and clinically evident cardiovascular disease, and completed patient enrollment in June 2015. Top-line results from the approximately 27,500-patient event-driven FOURIER study are anticipated in first quarter of 2017.

Repatha is approved in 43 countries, including the U.S., Japan, Canada and in all 28 countries that are members of the European Union. Applications in other countries are pending.

About Amgen Cardiovascular
Building on more than three decades of experience in developing biotechnology medicines for patients with serious illnesses, Amgen is dedicated to addressing important scientific questions to advance care and improve the lives of patients with cardiovascular disease, the leading cause of morbidity and mortality worldwide.(10) Amgen's research into cardiovascular disease, and potential treatment options, is part of a growing competency at Amgen that utilizes human genetics to identify and validate certain drug targets. Through its own research and development efforts, as well as partnerships, Amgen is building a robust cardiovascular portfolio consisting of several approved and investigational molecules in an effort to address a number of today's important unmet patient needs, such as high cholesterol and heart failure.

About Amgen
Amgen is committed to unlocking the potential of biology for patients suffering from serious illnesses by discovering, developing, manufacturing and delivering innovative human therapeutics. This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology.

Amgen focuses on areas of high unmet medical need and leverages its expertise to strive for solutions that improve health outcomes and dramatically improve people's lives. A biotechnology pioneer since 1980, Amgen has grown to be one of the world's leading independent biotechnology companies, has reached millions of patients around the world and is developing a pipeline of medicines with breakaway potential.

1. Repatha® U.S. Prescribing Information. Amgen.
2. World Health Organization. Quantifying Selected Major Risks to Health. In: The World Health Report 2002 - Reducing Risks, Promoting Healthy Life. Geneva. 2002:49-97.
3. Merck Manuals website. http://www.merckmanuals.com/professional/endocrine_and_metabolic_disorders/lipid_disorders/dyslipidemia.html. Accessed May 2016.
4. American Heart Association (2014). Why Cholesterol Matters. http://www.heart.org/HEARTORG/Conditions/Cholesterol/WhyCholesterolMatters/Why-Cholesterol-Matters_UCM_001212_Article.jsp. Accessed May 2016.
5. World Health Organization. Global Status Report on Noncommunicable Diseases 2014. Geneva, 2014.
6. Amgen Data on File.
7. Centers for Disease Control and Prevention. Vital signs: prevalence, treatment, and control of high levels of low-density lipoprotein cholesterol. United States, 1999-2002 and 2005-2008. MMWR. 2011;60(4):109-14.
8. National Human Genome Research Institute. Learning About Familial Hypercholesterolemia. http://www.genome.gov/25520184. Accessed May 2016.
9. Nordestgaard BG, Chapman MJ, Humphries SE, et al. Familial Hypercholesterolaemia is Underdiagnosed and Undertreated in the General Population: Guidance for Clinicians to Prevent Coronary Heart Disease. Eur Heart J. 2013;34:3478-3490.
10. World Health Organization. Cardiovascular diseases (CVDs) fact sheet. http://www.who.int/mediacentre/factsheets/fs317/en/. Accessed May 2016.

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