For patients whose cholesterol stays stubbornly high despite decades-old standard treatment, a new option arrived this week. The US Food and Drug Administration has approved Lipfendra, a once-daily pill from the drugmaker Merck, making it the first oral version of a potent class of cholesterol-lowering medicines that until now could only be injected.
The drug, known generically as enlicitide, is a PCSK9 inhibitor. It blocks a protein that governs how much low-density lipoprotein — LDL, or "bad" cholesterol — circulates in the blood, where excess levels drive the plaque build-up behind heart attacks and strokes. Existing PCSK9 inhibitors such as Amgen's Repatha and the Regeneron–Sanofi drug Praluent are effective but delivered by injection; a swallowable 20-milligram tablet lowers the practical barrier to reaching them.
The approval is aimed at a large and underserved group. Statins remain the most prescribed medicine in the United States, but they can cause side effects such as muscle aches and often fail to bring cholesterol down far enough. "Seventy percent of patients are not getting to their LDL goal," said Dr Paja Banka, a Merck clinical-development executive and pediatric cardiologist, who said the pill could be taken alongside a statin or, in some cases, on its own. Roughly one in four American adults has elevated LDL, according to the American Heart Association.
The clearance rested on two late-stage trials in Merck's CORALreef program. In patients with high cholesterol, the pill cut LDL by about 56 percent against placebo after 24 weeks; among those with an inherited form of the condition, heterozygous familial hypercholesterolaemia, the reduction reached roughly 59 percent — including in people already taking statins.
An oral route to a hard target
Convenience is the point. Cardiologists have long had drugs that drive LDL down sharply, but injections and cost have kept many patients from them. A daily pill, priced at $10.50 a day, could widen access, though real-world uptake will depend on insurance coverage and how many patients stay on a lifelong medication.
For Merck, the approval also broadens a portfolio heavily reliant on its cancer immunotherapy Keytruda, which faces the loss of key patent protection from 2028. For patients and their doctors, the more immediate significance is simpler: another, easier-to-take tool against one of the most common and preventable drivers of cardiovascular disease.