Amgen is hopeful that Phase III data will cement the validity of its recently approved widened label for cholesterol-lowering drug Repatha (evolocumab).
In August 2025, the US Food and Drug Administration (FDA) approved the use of Repatha in adults at increased risk for major adverse cardiovascular events (MACE) due to uncontrolled low-density lipoprotein cholesterol (LDL-C).
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Now, the VESALIUS-CV trial (NCT03872401) has proved its long-term efficacy in these patients after meeting both its primary endpoints, proving that Repatha can significantly reduce the risk of MACE in patients without a prior history of heart attack or stroke.
The primary endpoints were time to first occurrence of a composite of coronary heart disease (CHD) death, heart attack or ischemic stroke, and time to first occurrence of a composite of CHD death, heart attack, ischemic stroke or any ischemia-driven arterial revascularisation.
The trial enrolled more than 12,000 high-risk patients who were followed for 4.5 years. Of those enrolled, approximately 85% were maintained on a high-intensity or moderate LDL-C reducing therapy.
Amgen R&D executive vice-president Dr Jay Bradner said: “The benefit across endpoints and established safety profile underscore Repatha’s role as a cornerstone therapy in comprehensive lipid management.
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By GlobalData“Repatha is known as a highly effective LDL-C-lowering treatment and is now the first and only PCSK9 inhibitor shown to reduce cardiovascular events in high-risk adults without prior heart attack or stroke. These additional data demonstrate that Repatha has the potential to reach tens of millions more patients earlier in their journey, before a life-altering event occurs.”
Amgen has not yet released data from the trial; however, this is set to be presented at the American Heart Association Scientific Sessions on 8 November.
Repatha was first approved in the US for adults with high cholesterol who require further LDL-C reduction on 27 August 2015. This came after European approval, making it the first PCSK9 inhibitor to receive regulatory clearance worldwide.
Its long market presence means it is coming close to the end of its patent-protected period, due to end in 2030, opening up an opportunity for biosimilars.
GlobalData predicts this will dent Amgen’s market dominance, with peak sales for the drug reaching $3.87bn in 2029 before slowly dropping, with a 2031 sales forecast of $3.32bn.
GlobalData is the parent company of Clinical Trials Arena.
Repatha’s main market competition comes from Praluent (alirocumab), a PCSK9 inhibitor developed by Regeneron and Sanofi, which gained US approval before Repatha, but EU approval came later. Praluent sales are set to reach $880m in 2031, according to GlobalData.
After Praulent’s approval, Regeneron and Sanofi, and Amgen were embroiled in a patent dispute over the two therapies, with Amgen emerging victorious.
Amgen also faces competition from Novartis’ Leqvio (inclisiran), a long-acting small-interfering RNA (siRNA) that degrades PCSK9. GlobalData predicts sales will rise sharply, from $754m in 2024 to $3.98bn in 2031.
A number of LDL-C-lowering drugs have also recently shown promise, including LIB Therapeutics’ lerodalcibep, AstraZeneca’s AZD0780 and NewAmsterdam Pharma’s obicetrapib.
GlobalData predicts the total dyslipidemia (high cholesterol) market will reach $15.53bn across seven major markets (7MM: US, France, Germany, Italy, Spain, UK, and Japan) by 2032.
