FDA approves Merck daily cholesterol pill Lipfendra

The Food and Drug Administration has approved Merck’s Lipfendra, a once-daily cholesterol pill that brings a powerful class of LDL-lowering drugs to tablet form for the first time.

The drug is a PCSK9 inhibitor, a type of medication that until now had been available only as an injection. The FDA approved Lipfendra for people with hypercholesterolemia, or high levels of LDL cholesterol, often called “bad” cholesterol.

High LDL cholesterol can contribute to plaque buildup in the arteries, raising the risk of heart disease and stroke. About one in four U.S. adults have high LDL cholesterol, according to the American Heart Association. Heart disease is the leading cause of death in the United States, The Guardian reported.

For decades, statins have been the standard treatment for high cholesterol and are the most prescribed medications in the United States, according to The Guardian. But they can sometimes cause side effects such as muscle aches, and they do not always lower cholesterol enough for patients to meet recommended targets.

Dr. Paja Banka, Merck’s associate vice president for global clinical development and a pediatric cardiologist, said many patients remain above their LDL targets despite existing treatments.

“Seventy per cent of patients are not getting to their LDL goal,” she told The Guardian.

Banka said Lipfendra would be taken “in conjunction” with statins, though in some cases it could be used alone. She said side effects in trials appeared minimal, with no muscle aches, and “were like the placebo.”

Merck said Lipfendra should be available within weeks.

In one late-stage clinical trial, Merck found that Lipfendra lowered LDL cholesterol levels by up to 60% after 24 weeks. A second trial showed similar reductions among people with heterozygous familial hypercholesterolemia, a genetic condition that causes very high cholesterol from birth. Nearly all participants in the trials were already taking a statin, and many were also using ezetimibe, another cholesterol-lowering drug.

Lipfendra works by blocking PCSK9, a protein involved in the production of LDL cholesterol. Statins work differently, targeting an enzyme the liver uses to make cholesterol.

The first PCSK9 inhibitor, Amgen’s injectable Repatha, was approved in 2015. Regeneron and Sanofi also make an injectable PCSK9 inhibitor called Praulent.

The approval comes months after major medical groups, including the American Heart Association and the American College of Cardiology, issued new guidelines calling for more aggressive cholesterol treatment. The guidelines recommend LDL cholesterol below 100 mg/dL for people without major heart disease risk factors, below 70 mg/dL for people at high risk and below 55 mg/dL for people with heart disease or at very high risk of cardiovascular events.

Merck said Lipfendra will have a list price of $10.50 a day, or $315 for a 30-day supply. The company said it expects out-of-pocket costs to be lower for many patients.