Merck’s LIPFENDRA Receives FDA Approval for Cholesterol

Merck & Co. announced on Thursday that the U.S. Food and Drug Administration (FDA) approved LIPFENDRA, the first oral PCSK9 inhibitor designed to lower LDL cholesterol. The approval follows positive results from two pivotal Phase 3 trials, the CORALreef Lipids and CORALreef HeFH studies, which demonstrated significant reductions in LDL cholesterol levels.

Clinical Trial Results

In the CORALreef Lipids trial, patients taking LIPFENDRA achieved a 56% reduction in LDL cholesterol compared to a placebo at week 24. When adjusted for biologically impossible baseline LDL values, the reduction increased to 60%. In contrast, placebo patients experienced a 3% increase in LDL cholesterol. The trial also indicated reductions in other lipid measures, including a 54% decrease in non-high-density lipoprotein cholesterol and a 50% decline in apolipoprotein B.

The CORALreef HeFH trial, which focused on adults with heterozygous familial hypercholesterolemia, reported a 59% reduction in LDL cholesterol compared to placebo at week 24. The treatment group saw a decline of 58% from baseline, while placebo participants had a 3% increase. Additionally, the study recorded a 52% reduction in non-HDL cholesterol and a 48% decrease in apolipoprotein B.

Future Implications

Merck is currently conducting ongoing trials to evaluate whether LIPFENDRA can reduce cardiovascular morbidity and mortality. However, the company noted that it has not yet established whether the treatment lowers the risk of cardiovascular events or death. Following the announcement, Merck's shares rose 3.61% to $128.07, nearing its 52-week high of $130.29, according to Benzinga Pro data.