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A new pill to treat stubbornly high cholesterol has been approved by the Food and Drug Administration, a development that will make treatment more convenient for patients at high risk of cardiovascular disease and heart attack.
Merck, the manufacturer of the once-daily tablet, Lipfendra, announced the approval Thursday. The drug is the first oral form of a class of powerful cholesterol-fighting drugs called PCSK9 inhibitors. Merck said it will be available in a matter of weeks.
Lipfendra has been shown in clinical trials to reduce bad cholesterol, LDL-C, by 60 percent, with few side effects, Merck said in a news release. That effectiveness is in line with other drugs already on the market.
Merck is conducting an ongoing study to verify that reduction will translate to preventing strokes, heart attacks and death; the other drugs in the class have already been proven to improve those clinical outcomes.
Up to now, patients were required to self-inject PCSK9 drugs, the most common of which are monoclonal antibodies sold by Amgen and Regeneron/Sanofi. Now patients will have a more convenient option.
"This is a game changer," said Erin Michos, a cardiologist and professor at the Johns Hopkins School of Medicine. "I have a number of patients who are anxiously waiting for the oral one. They didn't want to go on an injection. I have to give them a call and say it's out now."
Who should be taking this drug?
These drugs are an important line of defense for people whose cholesterol won't come down enough with a statin alone.
Some patients do not respond well to a statin, which is the standard, low-cost treatment for high cholesterol. In many cases that is because of a condition called hypercholesterolemia, an inherited disorder.
Other patients may not be able to tolerate the side effects of a statin, which can cause muscle pain, weakness and headaches, as well as gastrointestinal issues.
"There are some patients who can't tolerate statins, or they can't tolerate statins at a high enough dose to achieve their LDL goals," Michos said. "The reality is there's a huge unmet need - lots of patients out there who are not at their goal."
And for people who don't want to inject themselves, the new pill presents an alternative.
What are the side effects?
Side effects affected relatively small numbers of people in the clinical trials for Lipfendra. Seven percent of subjects experienced diarrhea, compared with 2 percent on placebo. And 9 percent of participants suffered from dizziness, compared with 4 percent for the placebo. Merck said the people discontinuing treatment were similar in both arms of the trials.
How do these drugs work?
The drugs work by blocking a protein, PCSK9, that hinders the liver's ability to remove cholesterol from the blood.
The first drug in this class, Praluent, sold by Regeneron/Sanofi, was approved in 2015. Amgen's Repatha also was approved in 2015. Patients self-administered the drugs via a prefilled pen every two weeks.
Novartis won approval for its twice-yearly injectable RNA drug, Leqvio, in 2021. The FDA approved Lerochol, a once-monthly injection, in 2025.
A consideration with the new pill, which is a peptide drug: It must be taken in the morning, 30 minutes before eating food.
How much will it cost?
Merck said the Lipfendra price will be $10.50 per day, based on a 30-day supply, or $315 a month. The company said that is up to 50 percent cheaper than some of the existing drugs. It said it will offer patients coupons and also make it available on TrumpRx, President Donald Trump's direct-to-consumer sales channel.
On the drug website GoodRx, cash prices for the existing drugs, factoring in promotions and coupons, range from $199 to $532.
Patients with insurance, of course, will be responsible for a co-payment that will be far less. But insurance companies typically require use of a low-cost generic statin first, to see if that works. Getting prior authorization for the injectables has sometimes been difficult, Michos said, so she will be curious to see if the lower-cost pill loosens coverage decisions.
"In high-risk patients you don't have years to mess around trying every single statin and different statin doses," she said. "You have to move quickly and get them to goal quickly."
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