Can Amgen’s Repatha Stop Heart Disease Before It Starts?
Every 40 seconds, someone in the United States has a heart attack or stroke. Most of them don’t see it coming. One moment they’re at work or making dinner, the next they’re in an ambulance—or worse. For decades, doctors have struggled with this brutal fact: in most cases, the first symptom of heart disease is the event itself.
Now a new trial suggests that future might not be inevitable. Amgen’s cholesterol-lowering drug Repatha has done something no rival has managed—shown it can prevent heart attacks and strokes in people who have never had one before. The results, announced today, come from a sweeping 12,000-patient study called VESALIUS-CV. It tracked high-risk patients for more than four years and found that aggressively lowering cholesterol with Repatha can keep disaster at bay.
It’s a milestone. But as with all medical breakthroughs, the real question is what happens outside the controlled world of clinical trials.
Moving Beyond Statins
Statins have been the workhorses of heart disease prevention for decades. They’re cheap, effective, and have saved countless lives. Yet for all their power, statins don’t work for everyone. Many patients can’t lower their LDL cholesterol—the “bad” kind—enough to reach the safety zone. Others can’t tolerate high doses because of muscle pain or other side effects.
That’s why scientists turned to PCSK9 inhibitors, a new class of drugs that work differently. By blocking a protein that normally degrades cholesterol receptors in the liver, they supercharge the body’s ability to clear LDL from the bloodstream. In clinical terms, they can chop cholesterol by 60 percent or more.
Amgen launched Repatha in 2015, pitching it as the next big thing in cardiology. And it delivered—at least for patients who already had heart disease. A 2017 trial showed Repatha reduced repeat events like second heart attacks. The new question was whether it could work earlier, before the first strike. That’s what VESALIUS-CV set out to test.
What the Numbers Don’t Say
Here’s the catch: Amgen confirmed the trial hit its main targets but didn’t reveal by how much. That detail matters. A small relative benefit in a large, lower-risk population can look statistically significant without changing lives in practice.
Doctors use three measures to judge impact:
- Hazard ratio: the percentage risk reduction compared to a placebo.
- Absolute risk reduction: the actual difference in event rates.
- Number needed to treat: how many patients must get the drug to prevent one heart attack or stroke.
When Repatha was tested in patients with existing heart disease, doctors had to treat about 67 people for two years to prevent one event. In lower-risk groups, those numbers could balloon, making the drug less compelling—especially given its price tag.
Until the full results are presented at the American Heart Association’s annual meeting on November 8, no one knows whether Repatha’s win is a revolution or just a modest advance.
Why “What Counts” Counts
Trials often combine outcomes into what’s called a composite endpoint. That can include hard outcomes—heart attacks, strokes, and deaths—but also procedures like stenting or bypass surgery. The logic is simple: fewer procedures still benefit patients. But critics argue those numbers can be swayed by local practice patterns more than biology.
In Repatha’s earlier trials, much of the benefit came from reducing procedures, not deaths. If VESALIUS-CV repeats that story, the headlines will sound bolder than the reality. On the other hand, if the drug clearly prevents first-time heart attacks and strokes, it could rewrite prevention playbooks.
Who Really Needs It?
Not everyone faces the same danger. A middle-aged diabetic with sky-high LDL is worlds apart from someone younger with borderline cholesterol. The trial likely included more of the former than the latter to boost event rates. Subgroup analyses will reveal whether diabetics, statin-intolerant patients, or people with genetic cholesterol disorders see the most benefit.
That detail could shape guidelines for years. If doctors can identify who benefits most, insurers may be more willing to cover the drug, and patients may be more willing to take it.
Safety and the Long Game
For people who already feel sick, a drug that prevents another heart attack is an easy sell. For people who feel perfectly healthy, it’s harder. That’s why safety looms large in prevention. Patients won’t sign up for years of injections if the side effects are worse than the risk.
So far, PCSK9 inhibitors look safe. The most common problems—mild infections or sore injection sites—are manageable. Still, there have been faint signals about increased diabetes risk. The November results will show whether those blips matter or not.
Price: The Elephant in the Exam Room
Then there’s the issue of cost. Even after discounts, Repatha runs several thousand dollars a year. Insurers often force doctors and patients through exhausting approval processes—paperwork, appeals, and denials—before they’ll pay.
This has stifled adoption for nearly a decade. And that was for patients who already had heart disease. For people who haven’t yet had an event, convincing insurers to pay for preventive therapy will be an even steeper hill.
Competition on the Horizon
For now, Repatha has the spotlight. Rival PCSK9 drugs exist, but none have trial data in primary prevention. Novartis is testing Leqvio, a twice-yearly injection that could prove more convenient, but results are years away. Even more disruptive would be oral PCSK9 pills, currently in development. If they pan out, the entire treatment landscape could shift overnight.
Patients at the Center
Clinical trials capture heart attacks, strokes, and deaths. But they don’t measure what it feels like to give yourself an injection every two weeks, or to budget for a drug that costs more than a family vacation. Real-world adherence to preventive therapy is notoriously low. People start strong but fade over time, especially when they feel fine.
That’s why convenience and affordability may ultimately decide Repatha’s fate more than any hazard ratio. Medicine only works if people actually take it.
November 8: The Reveal
In a few weeks, the American Heart Association will present the full VESALIUS-CV data. Cardiologists will comb through the numbers, looking for answers to the questions that matter most: Did Repatha prevent heart attacks and strokes, or just delay procedures? Did certain groups see outsized benefit? Were there new safety concerns? How many patients stayed on the drug?
Only then will the medical community know if Repatha is ready to reshape prevention or simply refine it.
The Bottom Line: Repatha proves it’s possible to stop heart disease before it strikes. Whether that promise becomes reality will depend not just on biology, but on economics, policy, and the everyday choices of patients and doctors.