On Thursday, the U.S. Food and Drug Administration (FDA) approved Merck & Co Inc.’s (NYSE:MRK) LIPFENDRA (enlicitide) as an adjunct to reduce low-density lipoprotein cholesterol (LDL-C) in adults with hypercholesterolemia, including those with heterozygous familial hypercholesterolemia (HeFH).
The condition is characterized by the presence of high levels of cholesterol in the blood, particularly “bad” LDL cholesterol.
The company said the treatment is the first FDA-approved oral PCSK9 inhibitor and is designed as a once-daily pill to lower LDL cholesterol.
According to the company, the approval was supported by positive results from two pivotal Phase 3 studies in its CORALreef clinical program.
Phase 3 Trials Show Significant LDL-C Reductions
The FDA approval was based on findings from the Phase 3 CORALreef Lipids and CORALreef HeFH trials.
In the CORALreef Lipids study, patients receiving LIPFENDRA achieved a 56% reduction in LDL-C compared with placebo at week 24.
Under revised post-hoc data handling rules that excluded biologically impossible baseline LDL-C values, the reduction increased to 60%, while placebo patients recorded a 3% increase from baseline.
The trial also showed statistically significant reductions in other lipid measures associated with atherosclerotic cardiovascular disease risk. Non-high-density lipoprotein cholesterol fell by 54%, while apolipoprotein B declined by 50%.
CORALreef HeFH Results Support Approval
In the CORALreef HeFH trial involving adults with heterozygous familial hypercholesterolemia, LIPFENDRA reduced LDL-C by 59% compared with placebo at week 24.
From baseline, LDL-C declined by 58% in the treatment group, while placebo participants saw a 3% increase.
The study also reported a 52% reduction in non-HDL cholesterol and a 48% decline in apolipoprotein B compared with placebo.
Merck said an ongoing clinical trial is evaluating whether LIPFENDRA can reduce cardiovascular morbidity and mortality. The company noted that it has not yet been established whether the treatment lowers the risk of cardiovascular events or death.
MRK Price Action: Merck & Co shares were up 3.61% at $128.07 at the time of publication on Thursday. The stock is trading near its 52-week high of $130.29, according to Benzinga Pro data.
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