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Merck wins U.S. FDA approval for once-daily oral LDL-C drug LIPFENDRA

Merck wins U.S. FDA approval for once-daily oral LDL-C drug LIPFENDRA

The FDA cleared Merck's cholesterol pill for patients whose artery-clogging LDL remains elevated despite statins, marking the first noninjectable PCSK9 treatment in a market long dominated by costly shots.

Fact Check
Merck's official Businesswire press release explicitly confirms FDA approval of LIPFENDRA (enlicitide) as the first and only once-daily oral PCSK9 inhibitor to reduce LDL-C in adults with hypercholesterolemia. CNBC independently corroborates it is the first oral alternative to injectable PCSK9 inhibitors and was approved for patients including those on statins. The drug name, oral formulation, PCSK9 first-of-kind status, approving regulator (FDA), and indication all match the claim precisely.
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Summary

Merck's LIPFENDRA has won U.S. FDA approval for patients whose artery-clogging cholesterol remains high despite treatment with statins, the standard drugs used to cut heart attack risk. The pill, approved on Thursday, is the first noninjectable medicine that works by blocking PCSK9, a liver protein that limits the body's ability to clear cholesterol from the blood. Merck said the product will be marketed under the Lipfendra brand name. The approval was based on two studies in high-risk patients who added the pill to standard treatment including statins. In one 3,000-patient study, LDL cholesterol fell by more than 55% after six months, while a second study showed an average 59% reduction versus placebo. The effect declined only slightly over a year, and side effects including dizziness and diarrhea were similar to placebo. The pill must be taken on an empty stomach. The decision brings an oral option to a cholesterol-lowering class that has for more than a decade been led by injectable PCSK9 drugs from Amgen and others, where uptake has been constrained by high prices, insurance limits and restrained prescribing. Heart disease remains the leading cause of death in the United States, and high LDL is a major contributor to heart attacks and strokes.

Terms & Concepts
  • PCSK9: A liver protein that reduces the body's ability to remove LDL cholesterol from the blood.
  • PCSK9 treatment: A therapy that blocks PCSK9 to help lower LDL, or bad, cholesterol.
  • placebo: A dummy pill or inactive treatment used as a comparison in clinical studies.