Merck secures landmark FDA approval for cholesterol pill Lipfendra

USA—Merck has received approval from the US Food and Drug Administration (FDA) for Lipfendra, a once-daily oral medication designed to lower low-density lipoprotein (LDL) cholesterol, commonly known as “bad” cholesterol.

The approval marks the first oral PCSK9 inhibitor, offering patients an alternative to injectable therapies traditionally used to reduce LDL cholesterol levels.

Expanding treatment options for high cholesterol

For decades, statins have remained the standard treatment for high cholesterol and are among the most widely prescribed medicines in the United States.

While these drugs have significantly reduced the risk of heart attacks and strokes, they do not work equally well for every patient.

Some individuals fail to achieve recommended LDL cholesterol targets despite treatment, while others experience side effects such as muscle pain that limit long-term use.

According to Dr Puja Banka, Associate Vice-President for Global Clinical Development at Merck and a paediatric cardiologist, the approval of Lipfendra addresses a significant gap in cholesterol management.

“Seventy percent of patients are not getting to their LDL goal,” she said, highlighting the need for additional therapies that can help patients achieve healthier cholesterol levels.

Designed to complement statins

Merck said Lipfendra is primarily intended to be used alongside statin therapy to provide greater reductions in LDL cholesterol.

However, the company noted that the medicine may also be prescribed on its own for patients who cannot tolerate statins or who require an alternative treatment.

Clinical studies showed that Lipfendra produced few side effects.

Unlike statins, which are sometimes associated with muscle aches, the newly approved medicine demonstrated a safety profile comparable to placebo, according to Dr Banka.

She added that participants in clinical trials did not report increased muscle-related adverse effects.

The company expects Lipfendra to become commercially available within the coming weeks.

Addressing a major cardiovascular risk

Heart disease remains the leading cause of death in the United States, and elevated LDL cholesterol is a major contributor to atherosclerotic cardiovascular disease (ASCVD), in which fatty plaque builds up inside the arteries and restricts blood flow.

LDL cholesterol targets vary depending on an individual’s cardiovascular risk and medical history.

However, updated guidelines released in March by the American College of Cardiology and the American Heart Association recommend maintaining LDL cholesterol levels below 100 mg/dL for adults.

The guidelines further advise reducing LDL cholesterol to below 70 mg/dL for people at high risk of cardiovascular disease and below 55 mg/dL for individuals with established ASCVD who face a very high risk of future cardiac events.

Clinical trial data cited by The New York Times indicated that Lipfendra can lower LDL cholesterol to approximately 50–60 mg/dL or even lower in some patients.

Oral therapy may improve access

Until now, PCSK9 inhibitors have only been available as injectable medicines, which some patients find inconvenient or difficult to use consistently.

The availability of an oral tablet could encourage wider adoption of this class of cholesterol-lowering medicines by simplifying treatment.

Merck has set Lipfendra’s list price at US$10.50 per day, equivalent to more than US$300 per month, although the company said many patients are expected to pay lower out-of-pocket costs depending on their insurance coverage and reimbursement arrangements.