FDA approves first oral PCSK9 inhibitor enlicitide to lower LDL cholesterol

The U.S. Food and Drug Administration has approved enlicitide, the first oral tablet in the PCSK9‑inhibitor class, for treating elevated LDL (“bad”) cholesterol. The drug is intended for adults with primary hypercholesterolemia, heterozygous familial hypercholesterolemia, or those who cannot meet LDL targets despite statins or ezetimibe. By blocking the PCSK9 protein, enlicitide increases liver LDL‑receptor recycling, improving removal of LDL from the bloodstream. Until now, PCSK9 inhibition was available only as injectable biologics.

Clinical trials involving 3,207 patients on high‑dose statins showed an average LDL reduction of 56%, reaching up to 59% in patients with heterozygous familial hypercholesterolemia, with a safety profile comparable to placebo and the most common adverse events being diarrhea and dizziness. While the new pill expands options for LDL control, it does not address lipoprotein(a), a genetically determined cholesterol fraction linked to higher heart‑attack risk, which remains a focus of ongoing research.