RAHWAY, N.J.: Millions of people living with high cholesterol may soon have a new treatment option after the United States Food and Drug Administration (FDA) approved Merck's Lipfendra (enlicitide), a once-daily cholesterol pill that offers an alternative to injectable medicines used to lower LDL ("bad") cholesterol.
The approval marks a major milestone in cardiovascular medicine because Lipfendra becomes the first oral PCSK9 inhibitor to receive FDA approval, giving eligible patients a pill-based option in a treatment class that has, until now, been dominated by injections.
The decision is also strategically important for Merck as the pharmaceutical company works to diversify its portfolio ahead of the expected loss of patent protection for its blockbuster cancer immunotherapy Keytruda, which begins facing biosimilar competition from 2028 onward.
A new option for people with high cholesterol
High levels of low-density lipoprotein (LDL) cholesterol are a major risk factor for heart attacks, strokes and other cardiovascular diseases.
While statins remain the first-line treatment for lowering cholesterol, many patients either do not achieve recommended LDL targets or require additional therapy because they remain at high cardiovascular risk.
Until now, the most effective medicines beyond statins have largely been injectable PCSK9 inhibitors such as Repatha and Praluent.
Lipfendra introduces a new option by offering similar cholesterol-lowering therapy in a once-daily pill.
How does Lipfendra work?
Unlike statins, which reduce the liver's production of cholesterol, Lipfendra works by blocking the PCSK9 protein, allowing the liver to remove more LDL cholesterol from the bloodstream.
Because it targets cholesterol through a different biological pathway, Lipfendra can be used as an additional treatment for eligible patients whose LDL cholesterol remains elevated despite standard therapy.
Experts believe the availability of an oral treatment may also improve patient acceptance and long-term adherence among people who are reluctant to receive regular injections.
Clinical trials supported FDA approval
The FDA's decision was based on two Phase III clinical trials involving a broad range of patients with hypercholesterolemia, including individuals with familial hypercholesterolemia and patients already receiving statin therapy.
The studies showed that once-daily Lipfendra significantly lowered LDL cholesterol compared with placebo across multiple patient groups.
Researchers are continuing to evaluate whether these reductions in LDL cholesterol also translate into lower rates of heart attacks, strokes and cardiovascular deaths through an ongoing long-term cardiovascular outcomes study.
Why lowering LDL cholesterol matters
According to the American Heart Association, about one in four adults in the United States has elevated LDL cholesterol.
When LDL cholesterol builds up inside artery walls, it forms fatty plaques that narrow blood vessels, increasing the risk of coronary artery disease, stroke and peripheral arterial disease.
Because cardiovascular disease remains the world's leading cause of death, improving cholesterol management continues to be a major global public health priority.
Merck eyes growth beyond Keytruda
The approval also represents an important commercial milestone for Merck.
Its cancer immunotherapy Keytruda has become one of the world's highest-selling medicines, generating tens of billions of dollars in annual revenue. However, key patent protections are expected to begin expiring from 2028, prompting investors to closely monitor the company's next generation of growth products.
Industry analysts believe Lipfendra has significant commercial potential.
Some forecasts estimate annual peak sales exceeding US$5 billion, while others suggest the medicine could eventually generate tens of billions of dollars if widely adopted around the world.
Following the FDA announcement, Merck shares rose in premarket trading.
Fast-tracked through an FDA priority program
Lipfendra also benefited from the FDA Commissioner's National Priority Voucher programme, an initiative designed to accelerate the review of medicines considered important for public health or national priorities.
The expedited review reflects growing efforts to improve access to innovative therapies for chronic diseases that affect millions of people worldwide.
What happens next?
With FDA approval secured, physicians in the United States can now prescribe Lipfendra for eligible patients with hypercholesterolemia.
Cardiologists will now closely watch the results of ongoing cardiovascular outcomes trials to determine whether the cholesterol reductions seen with Lipfendra ultimately translate into fewer heart attacks, strokes and cardiovascular deaths.
If those benefits are confirmed, experts believe the once-daily pill could become one of the most significant advances in cholesterol management in recent years by expanding access to advanced LDL-lowering therapy without the need for injections.
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