FDA Expands Approval of Evolocumab to Include Patients at Increased Risk of MACE
The FDA removes the prior requirement for a patient who is already diagnosed with a cardiovascular disease.
The FDA extended the approval for evolocumab (Repatha) to include patients at an increased risk for major adverse cardiovascular events (MACE) due to uncontrolled low-density lipoprotein cholesterol (LDL-C). Because of the new update, the FDA removes the prior requirement for a patient who is already diagnosed with a cardiovascular disease.1
The FDA removes the prior requirement for a patient who is already diagnosed with a cardiovascular disease. | Image Credit: dima_oris - stock.adobe.com
"Far too many adults at risk of cardiovascular disease are not achieving their LDL-C goals, despite it being one of the most modifiable risk factors for a heart attack or stroke," Murdo Gordon, executive vice president of global commercial operations at Amgen, said in a news release.1 "This label update highlights the real-world need for additional treatment options for at-risk patients. Repatha is an effective therapy for reducing LDL-C, particularly in patients whose disease remains uncontrolled with statins or who cannot tolerate them."
Cardiovascular disease has been the leading cause of death in the United States since 1950, according to the CDC. From 2009 to 2012, the age-adjusted heart disease death rate decreased from 182.8 to 170.5 per 100,000, respectively, and then decreased slower in 2019 with 161.5 per 100,000 age-adjusted deaths. In 2019, the CDC reported a total of 659,041 deaths in 2019.2
High LDL-C is an established risk factor for cardiovascular diseases, including MACE—myocardial infarction, coronary artery disease death, or stroke. Current guidelines recommend lifestyle modifications that reduce total fat intake and dietary cholesterol, and when that is ineffective for patients, it is recommended to initiate statins. However, some patients could be intolerant to statins or experience adverse effects (AEs), including myopathy or gastrointestinal disorders.3
As part of the updated label for evolocumab, the FDA also expanded the use of the drug as a monotherapy for patients with homozygous familial hypercholesterolemia, and the drug continues to be recommended alongside diet and exercise to maintain high cholesterol.1
Evolocumab is a subcutaneous injection that is aimed at reducing LDL-cholesterol but has also been used to reduce the risk of death, heart attack, stroke, unstable angina requiring hospitalization, and heart surgery. It is a proprotein convertase subtilisin kexin type 9 inhibitor. Common AEs include back pain, injection site reaction, blurred vision, body aches or pains, chills, cough, dry mouth, headache, muscle aches, and gastrointestinal effects.4
In a study of patients with CAD and multivessel disease (MVD), investigators found that evolocumab reduced the rate of MACE for this patient population and for patients with CAD alone. However, the earlier and larger benefits were seen for those with MVD. The relative risk reduction of MACE for patients with MVD and treated with evolocumab was 23% compared with 12% for those without MVD.5
READ MORE: Cardiology Resource Center
Ready to impress your pharmacy colleagues with the latest drug information, industry trends, and patient care tips? Sign up today for our free Drug Topics newsletter.
REFERENCES
1. Repatha now indicated for adults at increased risk for major adverse cardiovascular events due to uncontrolled LDL-C. News release. Amgen. August 25, 2025. Accessed August 25, 2025. https://www.amgen.com/newsroom/press-releases/2025/08/repatha-now-indicated-for-adults-at-increased-risk-for-major-adverse-cardiovascular-events-due-to-uncontrolled-ldl-c
2. CDC. Heart disease deaths. August 5, 2024. Accessed August 25, 2025. https://www.cdc.gov/nchs/hus/topics/heart-disease-deaths.htm
3. Morgan CL, Durand A, McCormack T, Hughes E, Berni TR, Lahoz R. Risk of major adverse cardiovascular events associated with elevated low-density lipoprotein cholesterol in a population with atherosclerotic cardiovascular disease with and without type 2 diabetes: a UK database analysis using the Clinical Practice Research Datalink. BMJ Open. 2023;13(11):e064541. Published 2023 Nov 29. doi:10.1136/bmjopen-2022-064541
4. Mayo Clinic. Evolocumab (subcutaneous route). February 1, 2025. August 25, 2025. https://www.mayoclinic.org/drugs-supplements/evolocumab-subcutaneous-route/description/drg-20152627
5. McClintick DJ, O’Donoghue ML, De Ferrari MG, Ferreira JM, et al. Abstract 14566: long-term efficacy of evolocumab in patients with and without multivessel coronary artery disease. Circulation. Nov 2023. doi:https://doi.org/10.1161/circ.148.suppl_1.14566
Newsletter
Pharmacy practice is always changing. Stay ahead of the curve with the Drug Topics newsletter and get the latest drug information, industry trends, and patient care tips.
