FDA Approves Merck’s Lipfendra, First Oral PCSK9 Inhibitor for LDL Cholesterol

On July 16, the U.S. Food and Drug Administration granted approval for Lipfendra, a once-daily pill from Merck that targets the PCSK9 protein to lower low-density lipoprotein (LDL) cholesterol. The drug is indicated for adults with hypercholesterolemia, including those with the genetic condition familial hypercholesterolemia, and is intended to be used alongside a healthy diet, exercise, and other cholesterol-lowering therapies.

Merck wins FDA approval for oral LDL cholesterol drugFDA approves 1st-of-its-kind pill to reduce cholesterol

Lipfendra blocks PCSK9, a liver protein that normally prevents the body from clearing LDL from the bloodstream. In two late-stage trials, the pill reduced LDL levels by 56 % to 59 % compared with placebo, with consistent effects across diverse patient groups. The oral format offers an alternative for patients who cannot or prefer not to receive the injectable PCSK9 inhibitors that have been available from Amgen, Regeneron, and Sanofi.

FDA approves 1st-of-its-kind pill to reduce cholesterol

Merck has set the list price at $315 for a 30-day supply (approximately $10.50 per day). This represents a “meaningful discount” relative to the $500-$600 monthly cost of injectable PCSK9 drugs and the $400-$600 price range of branded statins. The company also plans to offer lower-cost options through the federal website trumprx.gov, which provides cash discounts on certain prescriptions.

Merck wins FDA approval for oral LDL cholesterol drug

Dean Yi, president of Merck Research Laboratories, called the approval “a pivotal moment” that adds a new therapeutic option for adults with high LDL-C. Dr. Jeffrey Berger, director of the Center for the Prevention of Cardiovascular Disease at NYU Langone Heart, emphasized that lowering LDL is a core preventive tool in cardiology. Dr. William Soliman, founder and CEO of the Accreditation Council for Medical Affairs, noted that the pill format could broaden adoption because many patients and physicians are more comfortable with pills than injections.

FDA approves 1st-of-its-kind pill to reduce cholesterol

• “Lowering LDL is one of the most effective tools we have in preventive cardiology because it directly targets a major driver of heart disease,” — Dr. Jeffrey Berger, NYU Langone Heart

FDA approves 1st-of-its-kind pill to reduce cholesterol

• “Ultimately, this approval expands our ability to personalize cholesterol treatment and help more people achieve the LDL levels that can better protect their cardiovascular health,” — Dr. Jeffrey Berger

FDA approves 1st-of-its-kind pill to reduce cholesterol

• “The oral format could broaden adoption because many patients and physicians are more comfortable with pills than injections,” — Dr. William Soliman, Accreditation Council for Medical Affairs

FDA approves 1st-of-its-kind pill to reduce cholesterol

• “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.” — Dean Yi, Merck Research Laboratories

Merck wins FDA approval for oral LDL cholesterol drug