Merck’s Lipfendra becomes world’s first oral PCSK9 treatment approved by FDA

Merck's Lipfendra (enlicitide) has become the world's first oral PCSK9 inhibitor approved by the U.S. FDA, offering a once-daily pill to lower LDL cholesterol in adults with hypercholesterolemia, including those with heterozygous familial hypercholesterolemia (HeFH).

The approval is based on two Phase 3 trials (CORALreef Lipids and CORALreef HeFH), where Lipfendra reduced LDL-C by about 56% to 59% compared with placebo at 24 weeks when added to statin therapy.

Lipfendra is a macrocyclic peptide that binds to PCSK9, blocking its interaction with LDL receptors, similar to injectable PCSK9 inhibitors but now available in oral form.

The drug is generally well tolerated; common side effects include diarrhea and dizziness in some patients, with safety profiles broadly comparable to placebo.

Lipfendra is positioned as an add-on to diet, exercise, and other cholesterol-lowering therapies, not a replacement, and is priced at about $315 per month, lower than typical injectable PCSK9 inhibitors.

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