Amgen's Repatha Shows Promise in Primary Prevention, Potentially Expanding Market for PCSK9 Inhibitors

Amgen has announced groundbreaking results from its Vesalius-CV trial, demonstrating that its cholesterol-lowering drug Repatha (evolocumab) met its primary endpoints in a large-scale study evaluating its use as a primary prevention therapy. This development could significantly expand the market for PCSK9 inhibitors and reshape cardiovascular disease prevention strategies.

Landmark Study Results

The Vesalius-CV trial, involving 12,000 participants with atherosclerosis or diabetes and elevated cholesterol levels, showed that Repatha reduced the risk of cardiovascular events or death in individuals who had never experienced a heart attack or stroke. This marks the first time a PCSK9 inhibitor has demonstrated efficacy in primary prevention, potentially paving the way for broader use of the drug.

Amgen reported that Repatha achieved "statistically and clinically significant" results on both main study objectives. However, the company has not yet disclosed the magnitude of the benefits observed. Detailed findings are scheduled for release at the American Heart Association meeting on November 8 in New Orleans and will be published in a medical journal.

Market Implications and Competition

Repatha, which generated $2.2 billion in revenue for Amgen in 2024, has fallen short of initial sales projections that once forecast tens of billions in annual sales. The drug currently faces challenges from insurers and has shown modest benefits in patients with established heart disease. However, this new data could substantially widen its use and boost sales.

The potential expansion comes at a crucial time for Amgen, as Repatha approaches the end of its patent-protected life. Biosimilar competition is expected in 2030, and pricing constraints from Medicare could further impact the drug's market potential as early as 2029.

Evolving Landscape of Cholesterol Management

While Repatha's new data is promising, the PCSK9 inhibitor market is becoming increasingly competitive. Merck & Co. is developing an oral PCSK9 blocker that has shown success in clinical testing earlier this year. Unlike Repatha, which requires injections every two weeks, Merck's drug is a daily pill. However, the need to fast after taking the oral medication could impact its practical use.

The FDA has recently expanded Repatha's approved use to include individuals at higher risk of major cardiovascular events, further solidifying its position in the market. As more data becomes available and new players enter the field, the landscape of cholesterol management and cardiovascular disease prevention continues to evolve, promising improved outcomes for patients at risk of heart disease.