From Breakthrough Science to Real-World Impact: Amgen’s Legacy in Cardiovascular Disease Care

Opening the Door to Next-Generation LDL-C Lowering Agents

In the early 2000s, researchers uncovered a unique genetic clue in people with a reduced risk of heart disease: specific mutations in the PCSK9 gene associated with lower levels of low-density lipoprotein cholesterol (LDL-C), or “bad” cholesterol.^1,2 That discovery opened the door to a new approach to LDL-C lowering.

The treatment of high LDL-C had long been addressed by one class of drugs: statins.³ However, some individuals experienced side effects from statin use.^4,5,6 Still others were unable to lower their LDL-C below recommended levels despite being on high or maximally tolerated doses.^4,5,6 Over the years, research has shown maintaining a low LDL-C level is crucial in reducing the risk of cardiovascular events like heart attack and stroke for people with established cardiovascular disease.^5,6

Having just one major class of lipid-lowering therapies drove the scientific community to search for other approaches.

The team at Amgen set out to develop a new therapy using insights from PCSK9 to help provide another treatment option for lowering LDL-C in addition to statins.⁷ The company spent nearly a decade researching and developing Repatha^® (evolocumab), a monoclonal antibody designed to help the body remove LDL-C from the blood.^8,9 It became one in a new class of cholesterol-lowering treatments known as PCSK9 inhibitors.⁷

Over 57,000 Patients in 51 Clinical Trials

In 2011, Amgen launched the PROFICIO clinical development program, a broad, multi-trial initiative to investigate the potential of Repatha across a wide spectrum of patients.¹⁰ In fact, over 57,000 patients have been studied in 51 clinical trials.¹¹ These studies have demonstrated Repatha’s ability to significantly lower LDL-C for patients with primary hyperlipidemia, heterozygous familial hypercholesterolemia (HeFH), and homozygous familial hypercholesterolemia (HoFH) and reduce the risk of major adverse cardiovascular events, like heart attack and stroke, in adults with established cardiovascular disease.^9,11

U.S. FDA Approval and Beyond

Results from the PROFICIO program helped pave the way for Repatha’s approval by the U.S. Food and Drug Administration (FDA) in August 2015, marking a new era with another therapeutic option for patients with high LDL-C.^10,12 For those who struggled to reach their LDL-C goals, Repatha was a breakthrough addition that could help transform the treatment landscape.^4,9 Fast forward to today, Repatha is approved in dozens of countries around the world.¹³

But approval was just the beginning. Amgen continued its research and, in 2017, published the FOURIER trial: a large cardiovascular outcomes study enrolling more than 27,000 adults with established CVD.^9,14 FOURIER set out to answer a critical question: could Repatha reduce the risk of cardiovascular events for these patients by lowering their LDL-C?^9,14 The answer was clear and compelling. The study found that adding Repatha to statin therapy significantly reduced the risk of composite cardiovascular events by 15% (time to first occurrence of MI, stroke, coronary revascularization, hospitalization requiring unstable angina, and CV death) (HR 0.85, 95% Cl, 0.79-0.92; [P<0.0001).]^9,* Studies like FOURIER have contributed to what is considered one of the most extensive and longest-running data sets in patients exposed to a PCSK9 inhibitor to date.¹⁵

Widening the Doorway: Making Repatha More Accessible

While scientific innovation is at the heart of every breakthrough, the team at Amgen understood that the true impact of a therapy is measured by the lives it touches.

In October 2018, Amgen took decisive action to help break down barriers for patients, announcing a 60% reduction in the U.S. list price of Repatha. For many patients, particularly those on Medicare, this made Repatha more accessible.¹⁶

As of August 2025, Repatha has been prescribed to more than 5 million^† patients worldwide and is the #1 covered PCSK9 inhibitor monoclonal antibody (PCSK9i mAb) in the U.S.^17,18,‡ Repatha is covered for approximately 96% of patients in the U.S. with established CVD,^‡ with over 80% paying $50 or less, regardless of insurance type.^18,19,§ Additionally, for about half of U.S. patients, there is no prior authorization required.²⁰ With fewer prior authorization requirements, many patients who are prescribed Repatha may be able to start treatment sooner.

“As we celebrate 10 years since the FDA approval of Repatha, we’re proud of the role Repatha plays in helping patients with established cardiovascular disease achieve their recommended LDL-C levels and reduce their cardiovascular risk,” said Nerissa Gomes, vice president and general manager of the cardiovascular business unit at Amgen. “The difference we can make for these patients drives our efforts every day.”

Through patient support programs, ongoing education, and working with healthcare providers and advocates, Amgen is matching life-changing innovation with real-world access a decade on—leading the way in making sure that groundbreaking therapies reach those who need them.

Footnotes

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IMPORTANT SAFETY INFORMATION

• Contraindications: Repatha is contraindicated in patients with a history of a serious hypersensitivity reaction to evolocumab or any of the excipients in Repatha. Serious hypersensitivity reactions including angioedema have occurred in patients treated with Repatha.

• Hypersensitivity Reactions: Hypersensitivity reactions, including angioedema, have been reported in patients treated with Repatha. If signs or symptoms of serious hypersensitivity reactions occur, discontinue treatment with Repatha, treat according to the standard of care, and monitor until signs and symptoms resolve.

• Adverse Reactions in Adults with Primary Hyperlipidemia: The most common adverse reactions (>5% of patients treated with Repatha and more frequently than placebo) were: nasopharyngitis, upper respiratory tract infection, influenza, back pain, and injection site reactions.

From a pool of the 52-week trial and seven 12-week trials: Local injection site reactions occurred in 3.2% and 3.0% of Repatha-treated and placebo-treated patients, respectively. The most common injection site reactions were erythema, pain, and bruising. Hypersensitivity reactions occurred in 5.1% and 4.7% of Repatha-treated and placebo-treated patients, respectively. The most common hypersensitivity reactions were rash (1.0% versus 0.5% for Repatha and placebo, respectively), eczema (0.4% versus 0.2%), erythema (0.4% versus 0.2%), and urticaria (0.4% versus 0.1%).

• Adverse Reactions in the Cardiovascular Outcomes Trial: The most common adverse reactions (>5% of patients treated with Repatha and more frequently than placebo) were: diabetes mellitus (8.8% Repatha, 8.2% placebo), nasopharyngitis (7.8% Repatha, 7.4% placebo), and upper respiratory tract infection (5.1% Repatha, 4.8% placebo).

Among the 16,676 patients without diabetes mellitus at baseline, the incidence of new-onset diabetes mellitus during the trial was 8.1% in patients treated with Repatha compared with 7.7% in patients that received placebo.

• Adverse Reactions in Pediatric Patients with HeFH: The most common adverse reactions (>5% of patients treated with Repatha and more frequently than placebo) were: nasopharyngitis, headache, oropharyngeal pain, influenza, and upper respiratory tract infection.

• Adverse Reactions in Adults and Pediatric Patients with HoFH: In a 12-week study in 49 patients, the adverse reactions that occurred in at least two patients treated with Repatha and more frequently than placebo were: upper respiratory tract infection, influenza, gastroenteritis, and nasopharyngitis. In an open-label extension study in 106 patients, including 14 pediatric patients, no new adverse reactions were observed.

Indications
Repatha is indicated:

• To reduce the risk of major adverse cardiovascular (CV) events (CV death, myocardial infarction, stroke, unstable angina requiring hospitalization, or coronary revascularization) in adults with established cardiovascular disease.
• As an adjunct to diet, alone or in combination with other low-density lipoprotein cholesterol (LDL-C)lowering therapies, in adults with primary hyperlipidemia, including heterozygous familial hypercholesterolemia (HeFH), to reduce LDLC .
• As an adjunct to diet and other LDL-C-lowering therapies in pediatric patients aged 10 years and older with HeFH, to reduce LDL-C.
• As an adjunct to other LDLC-lowering therapies in adults and pediatric patients aged 10 years and older with homozygous familial hypercholesterolemia (HoFH), to reduce LDLC.

The safety and effectiveness of Repatha have not been established in pediatric patients with HeFH or HoFH who are younger than 10 years old or in pediatric patients with other types of hyperlipidemia.

Please see full Prescribing Information.

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References