CHMP Backs Expanded Repatha Indication for Primary Prevention of First Heart Attack and Stroke
核心洞察
The EMA's CHMP adopted a positive opinion for Amgen's Repatha (evolocumab) to reduce cardiovascular risk in adults at high risk for ASCVD without prior heart attack or stroke.
The recommendation is based on the Phase 3 VESALIUS-CV trial of over 12,000 patients, which showed a 25% relative reduction in 3-P MACE and a 36% reduction in heart attack risk.
Repatha becomes the only PCSK9 inhibitor proven in a Phase 3 trial to significantly reduce the risk of a first major cardiovascular event.
Amgen announced on July 29, 2026, that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending an expanded indication for Repatha (evolocumab). The new indication covers adults with established or at high risk for atherosclerotic cardiovascular disease (搜索) (ASCVD) to reduce cardiovascular risk by lowering LDL-C levels, as an adjunct to correction of other risk factors. The recommendation specifically supports broader use of Repatha in patients who have not yet experienced a first heart attack or stroke.
"There remains a significant unmet need in Europe, where many patients at high cardiovascular risk are unable to achieve recommended LDL-C levels despite available lipid-lowering therapies," said Paul Burton, M.D., Ph.D., chief medical officer at Amgen. "The CHMP's positive opinion reflects the strength of Repatha's clinical evidence and brings us one step closer to making Repatha available to more patients who may benefit from it."
The VESALIUS-CV Trial: Landmark Evidence in Primary Prevention
The CHMP recommendation rests on results from the Phase 3 VESALIUS-CV trial, a double-blind, randomized, placebo-controlled, global clinical study that enrolled more than 12,000 patients. Participants had known ASCVD or high-risk diabetes, no history of heart attack or stroke, and elevated lipid levels — LDL-C ≥ 90 mg/dL, or non-HDL-C ≥ 120 mg/dL, or apolipoprotein B ≥ 80 mg/dL — despite treatment with the highest tolerated dose of statin and/or ezetimibe. The median baseline LDL-C was 122 mg/dL (IQR, 104–149 mg/dL). Patients were followed for a median of approximately 4.6 years.
Published in the New England Journal of Medicine in November 2025, the trial demonstrated that Repatha achieved a 25% relative reduction in the risk of the composite endpoint of coronary heart disease death, heart attack, or ischemic stroke (3-P MACE), and a 19% reduction in a broader composite that also included any ischemia-driven arterial revascularization (4-P MACE). Notably, evolocumab reduced the risk of heart attack by 36%. A consistent benefit was observed across all three endpoints.
In a lipid sub-study, adding evolocumab to a maximally tolerated dose of statin and/or ezetimibe substantially lowered LDL-C levels, with a median level of 45 mg/dL in the Repatha group compared to 109 mg/dL in the placebo group.
Setting a New Benchmark in Cardiovascular Risk Reduction
With this positive CHMP opinion, Repatha becomes the only PCSK9 inhibitor proven in a Phase 3 clinical trial to significantly reduce the risk of a first major cardiovascular event. The drug has been studied for 15 years across 51 clinical trials involving over 57,000 patients and has been used by more than 10 million patients globally since its initial approval in 2015.
Cardiovascular disease remains the leading cause of death worldwide. Recent research cited by Amgen shows that more than 99% of people who experience a first-time cardiovascular event have at least one traditional risk factor, including high LDL-C, which is one of the most modifiable risk factors for heart attack or stroke. While ESC/EAS guidelines recommend intensive LDL-C lowering based on cardiovascular risk, many high-risk patients remain above recommended LDL-C targets despite available lipid-lowering therapies.
Regulatory Path Forward
Repatha was initially approved in the European Union in 2015 for adults and pediatric patients with primary hypercholesterolemia (搜索) or mixed dyslipidemia, as well as to reduce cardiovascular risk in adults with established ASCVD. In August 2025, the U.S. Food and Drug Administration broadened the approved use of Repatha to include adults at increased risk for major adverse CV events due to uncontrolled LDL-C. The European Commission is expected to make a final decision on the expanded indication in the coming months.
If approved, the expanded indication would position Repatha as a primary prevention tool for high-risk patients across Europe, addressing a significant gap in cardiovascular care where many patients fail to reach guideline-recommended LDL-C targets with existing therapies.