FDA clears Merck pill for hard-to-treat LDL cholesterol
Lipfendra is the first approved oral drug to target PCSK9, a cholesterol pathway previously addressed with injectable medicines.
By Daniel Okafor · Business Editor
3 min read
The Food and Drug Administration has approved Merck’s Lipfendra, an oral cholesterol drug for patients whose LDL remains high despite treatment with statins. The decision could broaden use of a PCSK9-targeting approach that has been available for years mainly through costly injections.
Merck, based in Rahway, New Jersey, received clearance Thursday to sell the medicine for patients with artery-clogging cholesterol that is not adequately controlled by standard therapy, according to the FDA approval described by the Associated Press. The company will market the pill under the brand name Lipfendra.
Lipfendra is the first approved noninjectable medicine designed to block PCSK9, a protein in the liver involved in regulating how much cholesterol the body removes from the bloodstream. Injectable PCSK9 medicines from companies including Amgen have been on the market for more than 10 years, the AP reported, but use has been limited by high prices, insurance hurdles and uneven prescribing by physicians.
How the drug fits into cholesterol treatment
Statins remain the main treatment for lowering heart attack risk because they reduce part of the liver’s cholesterol production. Even at stronger doses, many patients need more LDL reduction to reach targets recommended in medical guidelines, according to the AP.
The FDA’s decision was based on two studies in high-risk patients who took Merck’s pill along with their usual treatment, including statins. In one trial involving 3,000 patients, Lipfendra users had LDL cholesterol reductions of more than 55% after six months, researchers found.
In a second study, patients taking the drug had an average LDL reduction of 59% compared with those given a placebo. Researchers reported that the effect eased only modestly over one year.
Side effects in the studies, including dizziness and diarrhea, were similar among patients taking Lipfendra and those taking placebo, according to researchers cited by the AP. Patients must take the pill on an empty stomach.
Fast FDA review
The FDA reviewed Lipfendra through a program meant to speed reviews for promising drugs viewed as serving the public interest. The AP reported that the pathway was established by then-FDA chief Dr. Marty Makary, who resigned in May after pressure from drug companies, patients and other outside groups.
High LDL cholesterol contributes to plaque buildup in arteries and is a major risk factor for heart attacks and strokes. Heart disease is the leading cause of death in the United States, according to the AP.
For healthy people, an LDL level of 100 is generally considered acceptable. After a diagnosis of high cholesterol or heart disease, doctors typically recommend lowering LDL to at least 70, with lower goals for patients at very high risk, the AP reported.
This story draws on original reporting from Fortune.