FDA on Friday approved Esperion Therapuetics' Nexletol, which is the first non-statin oral medication intended to lower cholesterol that FDA has approved in almost 20 years.
FDA approved Nexletol for patients who are genetically predisposed to have high cholesterol and patients who have heart disease. According to the Washington Post, Nexletol is a bempedoic acid and "is the first in a new class of drugs" intended to treat low-density-lipoprotein (LDL) cholesterol, or "bad" cholesterol.
The pill is designed as an alternative to statin pills, like Lipitor and Crestor, for the millions of patients who cannot tolerate statins because they experience harsh side effects from the drugs, such as muscle pain. Nexletol also can be used for patients who are taking statins but still are having trouble lowering their LDL cholesterol.
According to Robert Rosenson, director of cardiometabolic disorders for Mount Sinai Hospital, Nexletol targets enzymes in the liver, not the muscles, meaning they may not cause the same side effects as statins for some patients.
FDA recommended that patients using Nexletol should take the drug daily, in conjunction with the highest statin dose a patient can tolerate if possible and a healthy diet.
Esperion projects Nexletol could be effective for up to 18 million people in the United States and more than 100 million people in other countries. Tim Mayleben, president and CEO of Esperion, said the "approval delivers upon a commitment [the company] made to millions of patients for a new treatment alternative if they struggle with bad cholesterol."
Esperion said it expects to launch the drug in the United States in late March. The company did not announce a U.S. list price for the drug, put previously said it plans to price the drug at about $300 per month, the Associated Press reports.
Will providers prescribe Nexletol?
Experts are unsure whether Nexletol will be widely prescribed in the United States.
Christie Ballantyne, the cardiology chief at Baylor College of Medicine who has consulted for Esperion and helped test Nexletol, said the drug "is a nice alternative" to statins, but said statins likely will remain providers' first choice to help patients lower cholesterol.
But according to STAT+, Nexletol's approval will allow Esperion to test whether setting relatively lower prices for new heart drugs could help them succeed in the market.
STAT+ reports that "heart drugs have had a tough go of it in recent years," with multiple drugmakers seeing sales of new heart drugs plateau. For instance, Repatha and Praluent, PCSK9 cholesterol drugs introduced in 2015 by Regeneron and Sanofi, at first failed to pick up steam. The medicines originally cost $14,000 per year, leading health insurers to reject covering the drugs, even for the sickest patients. Eventually, companies reduced the drugs' costs to less than $6,000 and sales started to pick up—but the drugs still aren't widely used in the United States.
Ballantyne said although PCSK9s there "are highly effective, there are a lot of hassles with prior authorization and they often get denied."
But if Esperion prices Nexletol at about $300 per month, Nexletol would cost about one fifth of what other PCSK9s cost when they first were introduced in the U.S. market—and experts are questioning whether Nexletol's lower price will make it more appealing to insurers.
However, although Nexletol would be priced lower than PCSK9 inhibitors, it will be priced higher than generic statins. The generic version of the statin Lipitor, for instance, is priced at about $80 per year.
Michael Yee, an analyst at Jefferies, said investment banks estimate Nexletol will generate $35 million to $40 million in sales by the end of 2020, which is about the same as Praluent and Repatha generated in their first three quarters on the market. Yee said those sales targets mean providers would prescribe Nexletol 1,000 times by the send of 2020's second quarter and 2,500 times within the first six months the drug is on the market (Johnson, Associated Press, 2/22; McGinley, Washington Post, 2/21; Herper, STAT+, 2/21 [subscription required]).