FDA approves first cholesterol pill of its kind
The U.S. Food and Drug Administration (FDA) has approved enlicitide, a new oral PCSK9 inhibitor from Merck, to reduce low-density lipoprotein cholesterol (LDL-C) in adults with hypercholesterolemia. The approval covers patients presenting with heterozygous familial hypercholesterolemia (HeFH) as well.
This is first time an oral PCSK9 inhibitor has been approved by the FDA to lower LDL-C. All PCSK9 inhibitors on the market today are injectable—an oral option that does not require needles could make a significant impact on patient care.
Merck is selling enlicitide under the brand name Lipfendra. Patients take a 20-mg tablet of the drug once per day in addition to following a heart-healthy diet and regularly exercising.
“By harnessing the innovative science of PCSK9 inhibitors and novel macrocyclic peptide technology, Lipfendra was designed to significantly lower LDL-C in the form of a convenient once-daily pill,” Dean Y. Li, MD, PhD, president of Merck Research Laboratories, said in a statement. “This is a pivotal moment as we bring the first U.S. FDA-approved oral PCSK9 inhibitor to adults with high LDL-C, offering patients an important new option. We’re proud of our work with regulators on this rigorous and efficient review process.”
The FDA’s decision was largely based on two trials, CORALreef Lipids and CORALreef HeFH, that compared the drug to a placebo for 24 weeks. These studies confirmed that enlicitide could reduce LDL-C as well as other atherogenic lipoproteins linked to cardiovascular risk. An additional study is already underway exploring the drug’s potential to reduce the risk of cardiovascular morbidity and/or mortality.
“High LDL-C is a major risk factor for atherosclerotic cardiovascular disease, which is the leading cause of death globally,” Ann Marie Navar, MD, PhD, lead author of CORALreef Lipids and an associate professor of medicine at UT Southwestern Medical Center, said in the same statement. “In two Phase 3 trials, Lipfendra led to impressive reductions in LDL-C. Now, for the first time, patients have an oral PCSK9 inhibitor for LDL lowering.”
The long-term impact of this newly approved medication
There has been a substantial amount of excitement over the potential of enlicitide.
Jeffrey Berger, MD, director of the Center for the Prevention of Cardiovascular Disease at NYU Langone Heart, told Cardiovascular Business this approval represents "an important step forward" in terms of preventive cardiology.
"We have decades of evidence showing that lowering LDL-C, particularly in patients at high risk, leads to meaningful reductions in cardiovascular events," he said. "However, despite having multiple effective therapies available, many patients still do not reach their recommended LDL goals.
The approval of an oral PCSK9 inhibitor provides clinicians and patients with another powerful tool to lower LDL cholesterol. It expands our ability to personalize treatment based on a patient’s risk profile, preferences, and ability to access or tolerate existing therapies."
Berger added that he is looking forward to seeing additional data on the drug's ability to improve patient outcomes.
Michael has more than 19 years of experience as a professional writer and editor. He has written at length about cardiology, radiology, artificial intelligence and other key healthcare topics.