Merck’s Lipfendra can cut “bad” cholesterol by nearly 60 per cent, but further research is needed to confirm whether it prevents heart attacks and strokes
The United States Food and Drug Administration has approved the first once-daily oral PCSK9 inhibitor for adults with high cholesterol, offering patients a new alternative to injectable treatments.
The medicine, called Lipfendra, contains the active ingredient enlicitide and is made by the pharmaceutical company Merck. It has been approved for use alongside diet and exercise to reduce low-density lipoprotein cholesterol, commonly known as LDL or “bad” cholesterol.
High LDL levels can cause fatty deposits to build up inside the arteries, increasing the risk of heart disease, heart attacks and strokes.
Lipfendra works by blocking PCSK9, a protein involved in controlling the amount of LDL cholesterol in the blood. Existing PCSK9 inhibitors are mainly given by injection, making Lipfendra the first treatment of its kind to be available as a daily pill.
The approval could expand treatment choices for people whose cholesterol remains too high despite taking statins or other medicines. Some patients may also prefer a tablet because it is easier to take and does not require injections.
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Merck said the drug would have a US list price of $10.50 a day, equivalent to about $315 for a 30-day supply. The company hopes the price and convenience of the tablet will allow more patients to benefit from PCSK9 treatment.
Trials show major fall in LDL levels
The FDA’s decision was based on results from two late-stage clinical trials.
In the CORALreef Lipids study, involving 2,904 adults who needed further cholesterol reduction, Lipfendra lowered LDL levels by 56 per cent compared with a placebo after 24 weeks. A separate analysis recorded a reduction of up to 60 per cent.
A second trial involved 303 adults with heterozygous familial hypercholesterolaemia, an inherited condition that causes dangerously high cholesterol levels from birth. LDL fell by about 59 per cent compared with the placebo group after 24 weeks.
The wider trial found that the overall rate of side effects was similar among people taking Lipfendra and those receiving a placebo.
In the study involving people with inherited high cholesterol, diarrhoea was reported by seven per cent of Lipfendra users, compared with two per cent in the placebo group. Dizziness was recorded in nine per cent of patients taking the drug and four per cent of those receiving the placebo.
Statins remain central to treatment
The approval does not mean statins will be replaced. Updated guidance from the American Heart Association and the American College of Cardiology continues to describe statins as the foundation of cholesterol-lowering treatment. Additional medicines may be considered when lifestyle changes and statins do not bring LDL levels down sufficiently.
The guidelines recommend an LDL level below 70 milligrams per decilitre for people at high risk of suffering their first heart attack or stroke. Those with cardiovascular disease who face a very high risk of another serious event may be advised to reduce their LDL level below 55.
Lipfendra’s strong cholesterol-lowering effect could help more patients reach those targets, particularly those already receiving treatment but whose LDL remains above recommended levels.
However, one major question remains unanswered.
Although the drug has been shown to reduce cholesterol, it is not yet known whether it directly lowers the number of heart attacks, strokes or cardiovascular deaths. Merck is conducting a large outcomes trial involving more than 14,500 participants to examine those effects.
The approval therefore represents a significant development in cholesterol treatment, but patients should not change their medication without consulting a qualified healthcare professional.
