Amgen’s Repatha^® (evolocumab) Receives Approval In China As The First PCSK9 Inhibitor For The Treatment Of Homozygous Familial Hypercholesterolemia

Note: The following is an English translation of a news release issued in China.

SHANGHAI, CHINA (August 8th, 2018) — Amgen China today announced that its cholesterol-lowering agent Repatha^® (evolocumab) Injection has been approved by the National Drug Administration of China (CNDA, former CFDA). Repatha is the first PCSK9 inhibitor in China for the treatment of adults and adolescents over 12 years old with homozygous familial hypercholesterolemia (HoFH). Proprotein convertase subtilisin kexin type 9 (PCSK9) is a protein that reduces the liver's ability to remove low-density lipoprotein cholesterol (LDL-C) from the blood by binding to the low-density lipoprotein (LDL) receptor (LDLR). By inhibiting the binding of PCSK9 to LDLR, Repatha increases the number of LDLRs available to clear the LDL from the blood, thereby lowering LDL-C levels.

Repatha can be used as an adjunct to diet and other LDL-lowering therapies (e.g., statins, ezetimibe, and LDL apheresis) to further lower the LDL-C level. High LDL-C level is determined as an important risk factor for cardiovascular disease (CVD).^1,2

HoFH, an autosomal dominant genetic disease, is a rare disorder. The clinical manifestations of HoFH is markedly elevated LDL-C level since births, and subsequently an increased risk of developing overt atherosclerotic cardiovascular disease (ASCVD). If left untreated or poorly treated, most patients may suffer from angina or myocardial infarction during childhood or adolescence, which leads to premature death, before the age of 20 to 30.³ Other signs and symptoms of the disease may include extensive xanthomas and arcus corneae.

"It is estimated that one in 160,000 – 1,000,000 people is affected by HoFH⁴,” said Dr. Ge Junbo, chairman of Chinese Society of Cardiology, academician of the Chinese Academy of Sciences and professor at Fudan University Zhongshan Hospital. “As HoFH patients’ LDL-C level is several times higher than normal, and existing therapies have limitations, most patients fail to effectively control their LDL-C levels and are at continued risk of cardiovascular incidents. Evolocumab can significantly lower LDL-C levels by inhibiting PCSK9. Its timely approval in China not only brings new hope for HoFH patients, but also improves their quality of life."

According to the clinical data, Repatha significantly reduces LDL-C levels in HoFH patients who have not been able to lower their LDL-C through diet and other cholesterol-lowering medications. Compared with placebo (12-week treatment), Repatha reduced HoFH patients’ LDL-C levels by up to 31%.⁵ Its robust efficacy and favorable safety have been verified during a one-year study conducted for the long-term treatment of HoFH patients.⁶

“The approval of Repatha in China has the potential to truly improve the lives of patients living with this rare disease,” said Penny Wan, Head and General Manager of Amgen’s Japan and Asia Pacific Region. “With Amgen’s full commitment to serving patients and combating severe and chronic diseases, we look forward to bringing more innovative therapies to Chinese patients and contributing to the Healthy China Initiative.”

Repatha has been approved by the European Commission (EC) and the U.S. Food and Drug Administration (FDA), and is now available in more than 60 countries and regions, including countries in the European Union, United States, Australia and Japan.

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.⁷

Repatha is approved in more than 60 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

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.

Amgen established an affiliate in China in 2012, and has since opened its China Headquarters and Asia Research & Development Center in Shanghai. Amgen also has an office in Beijing, which is responsible for drug registration and clinical trials, with the aim to deliver its medicines to patients in China. For more information, visit www.amgen.cn.