Efficacy and Safety of Oral PCSK9 Inhibitor Enlicitide in Adults With Heterozygous Familial Hypercholesterolemia
JAMA 10.1001/jama.2025.20620November 09, 2025 at 5:00 PM EST
Does treatment with the oral proprotein convertase subtilisin/kexin type 9 inhibitor enlicitide lead to clinically meaningful reductions in low-density lipoprotein cholesterol in adults with heterozygous familial hypercholesterolemia?In this phase 3, randomized clinical trial including 303 adults with heterozygous familial hypercholesterolemia and stable background statin use, the percentage change in the level of low-density lipoprotein cholesterol at 24 weeks was clinically meaningful and significant in those treated with enlicitide (−58.2%) vs placebo (2.6%) (between-group difference, −59.4%).In adults with heterozygous familial hypercholesterolemia, enlicitide is an effective and well-tolerated treatment for lowering the level of low-density lipoprotein cholesterol.
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