Health · 17 Jul
FDA approves Lipfendra, the first PCSK9 cholesterol inhibitor available as a tablet
The FDA approved Lipfendra (enlicitide) as the first oral inhibitor of PCSK9, taken as a once-daily tablet as an adjunct to diet and exercise to lower LDL cholesterol in adults with hypercholesterolemia, including heterozygous familial hypercholesterolemia (HeFH). Until now, every PCSK9 inhibitor required injection; this approval adds a pill for people who still need more LDL reduction despite existing therapy. The drug was developed by Merck.
The approval is based on two randomized, double-blind, placebo-controlled trials involving 3,207 adults already on maximally tolerated statins. In the trial of people with established atherosclerotic cardiovascular disease or high risk for it (mean baseline LDL 96 mg/dL), Lipfendra produced an average 56% reduction in LDL at week 24 versus placebo; in the HeFH trial (mean baseline LDL 119 mg/dL), the reduction averaged 59%. Side effects were comparable to placebo in the first trial, with diarrhea and dizziness occurring more often in the second, and discontinuation rates were similar in both.
What this changes: patients who need PCSK9-class LDL reduction but dislike or struggle with injections now have a tablet option — though it is still an add-on, not a replacement for diet, exercise and statins. One thing to keep in mind: the approval endpoint is LDL reduction at 24 weeks, a surrogate, so long-term heart-attack-and-stroke outcome data for this pill are not yet part of the record. Ask your clinician whether it fits your treatment plan.