Amgen's Repatha Shows Promise in Cardiovascular Disease Prevention

Amgen's cholesterol-lowering drug Repatha (evolocumab) has demonstrated significant potential in preventing cardiovascular events in high-risk patients without prior heart disease, according to recent clinical trial results. This development could expand the drug's reach and solidify its position in the primary prevention market.

Phase III VESALIUS-CV Trial Results

The Phase III VESALIUS-CV study, involving over 12,300 high-risk patients, has yielded positive outcomes for Repatha. Amgen announced that the trial met its primary endpoints, showing a statistically and clinically significant reduction in the risk of major adverse cardiovascular events (MACE) compared to standard care alone.

Key findings from the study include:

• Participants had no prior history of heart attack or stroke but were considered high-risk due to elevated LDL cholesterol levels, diabetes mellitus, or peripheral arterial disease.
• The trial followed patients for a median duration of approximately 4.5 years.
• About 85% of participants were also receiving high-intensity or moderate therapy to manage their LDL cholesterol.

Dr. Jay Bradner, Amgen's Executive Vice President of R&D, stated, "These results mark an important milestone in the fight against cardiovascular disease, the leading cause of death worldwide."

Expanding Repatha's Market Reach

Repatha, a PCSK9 inhibitor first approved in 2015, has been steadily expanding its indications. Recent developments include:

• An FDA label expansion in August 2024, removing the requirement for a prior cardiovascular disease diagnosis and broadening its use to adults at increased risk of MACE due to uncontrolled LDL-C.
• The potential to reach tens of millions more patients earlier in their cardiovascular disease journey, as highlighted by the VESALIUS-CV trial results.

Analysts at Leerink Partners noted that while some physicians already prescribe Repatha off-label for primary prevention, the formal label expansion and new data "should help drive broader adoption" and "help broaden Repatha's reach into the preliminary prevention space."

Financial Performance and Future Outlook

Repatha has shown strong financial performance, with Amgen reporting:

• A 36% increase in sales to $2.22 billion for 2024.
• Second-quarter sales growth of 31% to $696 million in 2025 compared to the previous year.

However, challenges lie ahead for Amgen's heart blockbuster:

• Expected price controls under the Inflation Reduction Act (IRA) in 2029.
• Potential biosimilar competition in the U.S. market at the beginning of the next decade.
• Market exclusivity outside the U.S. may extend until May 2032, depending on patent outcomes.

As cardiovascular disease remains a significant health concern, with a heart attack or stroke occurring every 40 seconds in the U.S., Repatha's expanded use in primary prevention could play a crucial role in addressing this pressing medical need.