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Primary biliary cholangitis (PBC) — previously known as primary biliary cirrhosis — is a chronic (long-term) liver condition that can lead to liver failure if not treated early. PBC happens when the immune system mistakenly attacks the small bile ducts inside the liver. As bile builds up, it can cause liver damage, including fibrosis (scarring) and, in later stages, cirrhosis (severe liver scarring).
There’s no cure for PBC, but thanks to the latest research, new treatment options are now available. These newer medicines may slow liver damage and help with symptoms that have been difficult to treat, particularly fatigue (extreme tiredness that doesn’t go away with rest) and pruritus (persistent itching). This article explores the latest treatments for PBC, how they work, and what side effects to look out for.
For many years, doctors used a drug called ursodeoxycholic acid (UDCA) — also called ursodiol — to treat PBC. It’s a naturally occurring bile acid that helps promote bile flow and secretion, reduce toxic bile acids, and decrease inflammation and damage in the liver. UDCA can lower liver enzymes like alkaline phosphatase (ALP), an enzyme that’s released by irritated bile ducts.
However, UDCA doesn’t work for everyone. Some people don’t respond to treatment, even after taking it for a year. Others can’t tolerate it due to side effects, such as hair loss, weight gain, and diarrhea.
Another drug — obeticholic acid (Ocaliva) — was later approved by the U.S. Food and Drug Administration (FDA) in 2016. It helps lower bile acid levels by turning on a protein called Farnesoid X receptor (FXR) in the liver. It’s used alone or in combination with UDCA. The downside is that it can make itching worse, and it’s not safe for people with cirrhosis. In rare cases, it has caused serious liver injury, even in people without advanced liver disease. The FDA recommends avoiding obeticholic acid in people with decompensated cirrhosis, and those taking it should be closely monitored.
Thankfully, new treatment options are now available. These newer medicines offer hope for people who haven't responded well to older drugs.
In 2024, the FDA approved two new drugs for treating PBC: seladelpar (Livdelzi) and elafibranor (Iqirvo).
Seladelpar is a drug that helps reduce bile buildup and inflammation in the liver. Taken by pill once a day, it works by turning on a special protein in the liver called peroxisome proliferator-activated receptor delta (PPAR-delta). This protein helps the liver break down fats and bile more efficiently.
In clinical studies, seladelpar lowered ALP levels and significantly reduced itching. About 25 percent of people saw their ALP levels return to normal. During the study, no serious treatment-related adverse effects were reported.
However, the most common side effects included:
The FDA approved the use of seladelpar in combination with UDCA. It may also be used on its own for people who don’t respond to or can’t tolerate UDCA because of the side effects. Seladelpar shouldn’t be used in people who have decompensated cirrhosis, a more advanced form of liver damage with complications.
Elafibranor also helps the liver manage bile and fat better. It turns on two proteins: PPAR-alpha and PPAR-delta. These help reduce bile acid buildup and liver inflammation.
Clinical trials showed that more than half of the people taking elafibranor had big drops in ALP levels. Some even reached normal levels.
Elafibranor is taken by pill at the same time every day, with or without food. The most common side effects are:
It can be taken on its own or together with UDCA. It’s also an option for people who haven’t had success with UDCA. Like seladelpar, elafibranor isn’t suitable for people with decompensated cirrhosis. Your doctor will monitor your liver and muscle enzyme levels during treatment to help ensure the medication is working safely and not causing side effects.
There are other treatments for PBC currently being tested in clinical trials, too. These drugs aren’t yet FDA-approved, but may become treatment options soon.
Setanaxib is a new drug currently being tested for PBC. It may help slow down or prevent fibrosis. It’s a NOX inhibitor, which means it works by blocking an enzyme called nicotinamide adenine dinucleotide phosphate hydrogen (NADPH) oxidase. This enzyme causes harmful inflammation in the liver. Blocking it may reduce liver damage and improve liver function over time.
In early-stage clinical trials, people with PBC who took setanaxib had lower ALP levels. Some even reported they felt less fatigued.
Setanaxib is still being studied, so it’s too early to say who might benefit most from it. It’s been well tolerated so far, but some people in trials had mild side effects, like a headache or upset stomach. Once the studies are published, we’ll know more about how well it works and who it may be used for.
Tropifexor may help treat PBC by turning on the FXR protein. This protein helps control how the liver makes and moves bile acids. By using tropifexor, the liver may help reduce bile acid production and prevent bile buildup, which can damage the liver over time.
Tropifexor isn’t made from bile acid, so it may cause less itching than older drugs like obeticholic acid, which also target FXR.
In early clinical trials, participants taking tropifexor showed improvements in gamma-glutamyl transferase (GGT) levels — a sign of improved liver health. So far, the drug is considered to be safe, although some people reported mild side effects like itching or small increases in certain liver enzymes. Research is still underway to confirm how well it works and how safe it is in the long run.
Linerixibat is a type of medication called an ileal bile acid transporter (IBAT) inhibitor, and it’s currently being studied to help people with PBC who experience itching. As noted, itching is very common in people with PBC and in those who are currently taking traditional PBC treatments, like UDCA and obeticholic acid. This itching can be very hard to treat and can greatly affect your quality of life and sleep.
Linerixibat works in the gut, not the liver. It blocks the ileal bile duct transporter protein, which normally helps the body reabsorb bile acids in the intestines. By interfering with this process, more bile acids are removed through the stool (poop) instead of going back into the body. This helps lower the amount of bile acids that may be contributing to itching.
In clinical trials, people who took linerixibat had less itching than those who took a placebo (drug with no active ingredients). The most common side effect was diarrhea. Linerixibat is still being tested, but so far, the results look promising.
Your doctor will look at your blood tests, symptoms, imaging, and signs of advanced liver disease and liver decompensation when deciding if you need a new treatment. These decisions will vary from person to person. However, you may be a good candidate if:
Most new drugs can be added to your current UDCA treatment. However, in some cases, they may be used on their own. Even if your doctor decides that you’re not a good candidate for these medicines, there may be new supportive therapies that can help you manage your symptoms better.
To gauge how well your treatment is working, your doctor will ask about your symptom improvements and run blood tests. The main test is for ALP. However, they’ll also check bilirubin levels because high levels can mean the liver isn’t working well. Other blood tests might check aspartate aminotransferase (AST) and alanine aminotransferase (ALT) to get a better picture of your overall liver health. Doctors also use a test called FibroScan, which is similar to an ultrasound, to look for liver scarring. There are also specialized lab tests that can check for the level of liver scarring.
By joining a clinical trial, you can help doctors test new treatments for PBC. If you want to take part in one, talk to your liver specialist or your healthcare team. They can help you find out which ones you qualify for that are recruiting in your area. You can also search online at clinicaltrials.gov. Search “primary biliary cholangitis” to see open studies.
On myPBCteam, the social network for people with primary biliary cholangitis and their loved ones, members come together to ask questions, give advice, and share their tips with others who understand life with primary biliary cholangitis.
What new treatment for PBC are you interested in learning more about? Have you taken either of the newly approved PPAR agonists, elafibranor or seladelpar? Share your experiences in the comments below, or start a conversation by posting on your Activities page.
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