Amgen (NASDAQ:AMGN) today announced that the U.S. Food and Drug Administration (FDA) has approved Repatha® (evolocumab) as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C)-lowering therapies for the treatment of pediatric patients aged 10 years and older with heterozygous familial hypercholesterolemia (HeFH) to reduce LDL-C.
| THOUSAND OAKS, Calif., Sept. 24, 2021 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced that the U.S. Food and Drug Administration (FDA) has approved Repatha® (evolocumab) as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C)-lowering therapies for the treatment of pediatric patients aged 10 years and older with heterozygous familial hypercholesterolemia (HeFH) to reduce LDL-C. HeFH is an inherited, genetic condition with a prevalence of one in 250 people worldwide.1 High levels of LDL-C starting at birth accelerate the development of atherosclerotic cardiovascular disease, leading to an overall increased risk of cardiovascular events, including heart attack and other vascular conditions, at an earlier age.2 Children with familial hypercholesterolemia (FH) can be normal weight, have a good diet, exercise enough and still have high LDL-C.2,3 “The approval of Repatha for pediatric patients with FH represents a much-needed adjunct treatment option for these children with genetically high cholesterol who are unable to manage their high LDL-C with other lipid-lowering agents alone,” said David M. Reese, M.D., executive vice president of Research and Development at Amgen. “This milestone further reinforces the safety profile of Repatha and aligns with Amgen’s commitment to addressing the unmet needs of the high-risk cardiovascular community.” The approval is based on the HAUSER-RCT Phase 3b study evaluating the safety and efficacy of Repatha in pediatric patients, 10 - 17 years of age, with HeFH. Monthly treatment with Repatha reduced LDL-C by mean 38% (95% CI: 45%, 31%; p < 0.0001) from baseline compared to placebo, meeting its primary endpoint.4 Reductions in LDL-C were observed by the first post-baseline assessment at the Week 12 time point and were maintained throughout the trial.4 Patients treated with Repatha had improved secondary lipid parameters from baseline in comparison to placebo, including a 35% (CI: 42%, 28%) reduction in non-high-density lipoprotein cholesterol (non-HDL-C) at week 24, a 27% (CI: 32%, 21%) reduction in total cholesterol at week 24 and a 32% (CI: 39%, 26%) reduction in apolipoprotein B (ApoB) at week 24.5 No new safety risks were identified.5 The most common treatment-emergent adverse events (>5% of patients treated with Repatha and occurring more frequently than placebo) included nasopharyngitis, headache, oropharyngeal pain, influenza and upper respiratory tract infection.5 “As pediatric FH is an under-recognized condition that can lead to premature coronary artery disease, it’s critically important to have additional treatments that can significantly lower cholesterol,” said Katherine Wilemon, founder and chief executive officer at The FH Foundation. The FDA also approved Repatha as an adjunct to other LDL-C lowering therapies for the treatment of homozygous familial hypercholesterolemia (HoFH) for younger pediatric patients. Repatha was already approved for treatment in HoFH patients aged 13 and older and is now available as a treatment for patients aged 10 and older. About Familial Hypercholesterolemia About HAUSER-RCT Study Design About Amgen in the Cardiovascular Therapeutic Area About Amgen Amgen focuses on areas of high unmet medical need and leverages its biologics manufacturing 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 the world’s largest independent biotechnology company, has reached millions of patients around the world and is developing a pipeline of medicines with breakaway potential. For more information, visit www.amgen.com and follow us on www.twitter.com/amgen. About Repatha® (evolocumab) Repatha is approved in 75 countries, including the U.S., Japan, China and in all 27 countries that are members of the European Union. Applications in other countries are pending. Indications Repatha® is indicated:
Important Safety Information
Please contact Amgen Medinfo at 800-77-AMGEN (800-772-6436) or 844-REPATHA (844-737-2842) regarding Repatha availability or find more information, including full Prescribing Information, at www.amgen.com and www.Repatha.com. Amgen Forward-Looking Statements No forward-looking statement can be guaranteed and actual results may differ materially from those we project. Discovery or identification of new product candidates or development of new indications for existing products cannot be guaranteed and movement from concept to product is uncertain; consequently, there can be no guarantee that any particular product candidate or development of a new indication for an existing product will be successful and become a commercial product. Further, preclinical results do not guarantee safe and effective performance of product candidates in humans. The complexity of the human body cannot be perfectly, or sometimes, even adequately modeled by computer or cell culture systems or animal models. The length of time that it takes for us to complete clinical trials and obtain regulatory approval for product marketing has in the past varied and we expect similar variability in the future. Even when clinical trials are successful, regulatory authorities may question the sufficiency for approval of the trial endpoints we have selected. We develop product candidates internally and through licensing collaborations, partnerships and joint ventures. Product candidates that are derived from relationships may be subject to disputes between the parties or may prove to be not as effective or as safe as we may have believed at the time of entering into such relationship. Also, we or others could identify safety, side effects or manufacturing problems with our products, including our devices, after they are on the market. 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An outbreak of disease or similar public health threat, such as COVID-19, and the public and governmental effort to mitigate against the spread of such disease, could have a significant adverse effect on the supply of materials for our manufacturing activities, the distribution of our products, the commercialization of our product candidates, and our clinical trial operations, and any such events may have a material adverse effect on our product development, product sales, business and results of operations. We rely on collaborations with third parties for the development of some of our product candidates and for the commercialization and sales of some of our commercial products. In addition, we compete with other companies with respect to many of our marketed products as well as for the discovery and development of new products. Further, some raw materials, medical devices and component parts for our products are supplied by sole third-party suppliers. Certain of our distributors, customers and payers have substantial purchasing leverage in their dealings with us. The discovery of significant problems with a product similar to one of our products that implicate an entire class of products could have a material adverse effect on sales of the affected products and on our business and results of operations. Our efforts to collaborate with or acquire other companies, products or technology, and to integrate the operations of companies or to support the products or technology we have acquired, may not be successful. A breakdown, cyberattack or information security breach could compromise the confidentiality, integrity and availability of our systems and our data. Our stock price is volatile and may be affected by a number of events. Global economic conditions may magnify certain risks that affect our business. Our business performance could affect or limit the ability of our Board of Directors to declare a dividend or our ability to pay a dividend or repurchase our common stock. We may not be able to access the capital and credit markets on terms that are favorable to us, or at all. The scientific information discussed in this news release related to our product candidates is preliminary and investigative. Such product candidates are not approved by the U.S. Food and Drug Administration, and no conclusions can or should be drawn regarding the safety or effectiveness of the product candidates. CONTACT: Amgen, Thousand Oaks References 1 The FH Foundation. Heterozygous vs Homozygous FH. https://thefhfoundation.org/heterozygous-vs-homozygous-fh. Accessed August 2021.
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Company Codes: NASDAQ-NMS:AMGN |
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