Federal regulators on July 16 approved a first-of-its-kind pill that targets cholesterol levels.
Food and Drug Administration officials cleared enlicitide, or Lipfendra, from Merck. The pill is designed to be taken once per day and reduce levels of cholesterol, a waxy substance that is crucial to the body’s function but at high levels can restrict blood flow and lead to problems such as heart attacks.
Efficacy and safety data, regulators said, were demonstrated in two randomized, double-blind, placebo-controlled trials in about 3,200 adults who have a condition called severe hypercholesterolemia, which is characterized by high amounts of low-density lipoprotein, or so-called bad cholesterol. The adults were also taking statins.
Enlicitide recipients in one of the trials saw a 56 percent drop in low-density lipoprotein baseline levels when compared to the placebo arms, and the other trial saw a 59 percent drop.
Adverse events were mostly similar across the arms, except that certain events such as diarrhea and dizziness occurred more frequently among drug recipients in one of the trials.
The FDA said the pill is meant for adults who have high cholesterol, including people who have inherited high cholesterol through heterozygous familial hypercholesterolemia.
About 25 percent of adults in the United States have high low-density lipoprotein levels, according to the American Heart Association.
The lipoprotein is known as bad cholesterol because it can build up over time in one’s arteries.
The association and other groups said in March that adults who have levels of 70 and above should work to lower the levels through lifestyle changes and, possibly, drugs such as statins.
Statins and other options already exist to treat high cholesterol, but Lipfendra is the first oral drug to block proprotein convertase subtilisin/kexin type 9 (PCSK9), a protein that regulates the number of cholesterol receptors in the body.
“This is a pivotal moment as we bring the first U.S. FDA-approved oral PCSK9 inhibitor to adults with high [low-density lipoprotein], offering patients an important new option,” Dr. Dean Li, president of Merck Research Laboratories, said in a statement. “We’re proud of our work with regulators on this rigorous and efficient review process.”
Katherine Wilemon, CEO of the Family Heart Foundation, said in a statement that the group was encouraged by the approval of the new option for adults who need to reduce their cholesterol levels.
The price of Lipfendra has not been released.
Merck did not respond to a request for comment by publication time.
AstraZeneca is testing its own daily pill targeting low-density lipoprotein in a phase 3 clinical trial, after earlier testing showed favorable results.