People with high cholesterol now have a new option that comes in pill form. The Food and Drug Administration approved Lipfendra, a once-daily oral medication for adults with high LDL cholesterol. According to Healthline, it is the first PCSK9 inhibitor in its class that does not require injections. The three other PCSK9 inhibitor treatments currently available all involve shots.
LDL cholesterol, often called bad cholesterol, builds up in the arteries and raises the risk of heart attack, stroke, and artery disease. Most people who need cholesterol medication start with a statin, but statins do not always work well enough on their own. Lipfendra can be taken alongside a statin or instead of one, and it works through a different mechanism.
Cardiologist Yu-Ming Ni, a lipidologist at MemorialCare Heart and Vascular Institute at Orange Coast Medical Center in Fountain Valley, California, said the oral format matters to patients. "I have a lot of people who are hesitant to even start because they don't want to give themselves injections," Ni told Healthline. "Now you've got this oral version. It's going to be a little bit easier for people to take a pill every day."
Anurag Mehta, a preventive cardiologist at Emory Healthcare and assistant professor of medicine at Emory University School of Medicine, explained how the two drug types work together. He said Lipfendra and statins target "two different biological pathways" to reach "the same end result" of lower LDL cholesterol. For patients whose statin is not doing enough on its own, "those patients would be candidates for this new medicine," Mehta said.
The FDA's approval rests on two clinical trials that together enrolled more than 3,200 adults already taking the highest statin dose they could tolerate. After 24 weeks, Lipfendra lowered LDL cholesterol by about 56% in the main study. In a second study focused on people with an inherited form of high cholesterol, the reduction was about 59%. Ni described those numbers as "quite impressive."
The trials measured how much the drug lowers cholesterol, not whether it prevents heart attacks or strokes directly. A larger trial testing that question is still underway. The FDA told Healthline it accepts a drop in LDL as a validated stand-in for lower cardiovascular risk when approving cholesterol drugs.
The prescribing information for Lipfendra states that it can be used as an additional therapy alongside diet and exercise for adults with high LDL cholesterol. Treatment decisions remain between patients and their doctors.