Primary Biliary Cholangitis (PBC): What to Know About Treatments

Primary biliary cholangitis (PBC) is a rare autoimmune disease in which the body attacks and progressively destroys the bile ducts in the liver. This leads to bile buildup that causes inflammation and scarring (fibrosis) until the scar tissue eventually replaces healthy liver tissue. Without treatment, the disease can progress to cirrhosis and liver failure. At that point, your liver can no longer clear toxins from your blood, make the digestive fluid bile, or do its many other important jobs.

In 1997, the FDA approved the first treatment for this condition, ursodeoxycholic acid (UDCA). Obeticholic acid (OCA) was FDA-approved in 2016. Seladelpar (Livdelzi) and elafibranor (Iqirvo) are the newest PBC drugs to be granted FDA approval.

There's no cure for PBC, but treatments like these can help to slow liver damage and reduce symptoms like itchy skin, tiredness, and pain.

What Are the Treatments for PBC?

These medicines help reduce scarring and improve symptoms and liver function. Some PBC treatments bring down inflammation in the liver. The goal of treatments is to slow the disease as much as possible and relieve symptoms. These are the approved medicines for PBC:

Researchers are studying more new treatment options in clinical trials, including bezafibrate and fenofibrate, which are in the same class of drugs as Iqirvo. They help to reduce inflammation in the body and have shown promise for improving PBC in people who haven't responded to UDCA.

Understanding how each of these medicines work, how you take them, and what side effects they might cause can help you have a more informed discussion with your doctor. Most people with PBC take UDCA first. If you don't see enough improvement on UDCA, your doctor might add or switch you to Iqirvo, Livdelzi, or Ocaliva.

What Is UDCA?

UDCA, also called ursodiol (Actigall, Urso), is a bile acid that is made from dried bear bile. Bile is a digestive fluid that your liver makes. Bile acids help your body break down fat and certain vitamins from foods so that your intestines can absorb them more easily. Your liver makes bile acids from cholesterol.

Doctors have been prescribing UDCA to treat liver disease for more than 100 years. It's been approved to treat PBC since 1997.

When you have PBC, bile gets stuck inside your bile ducts and damages your liver. UDCA treats this condition by:

Clearing bile from your liver
Protecting your liver cells from damage
Reducing inflammation in your liver
Calming your immune system to prevent it from damaging your liver

UDCA comes as a tablet that you take once or twice a day.

How Effective is UDCA?

Studies show that this medicine can slow disease progression and delay cirrhosis, especially if you start taking it in the early stages of PBC. UDCA also helps some people avoid the need for a liver transplant. Many people who take this medicine have improved liver function tests and are less likely to need a liver transplant.

What are the side effects of UDCA?

Studies have found that side effects of UDCA are relatively rare. But the drug may cause side effects like these:

Weight gain
Hair loss
Diarrhea
Nausea
Vomiting

Is UDCA safe?

Long-term research has shown that UDCA is well tolerated with few long-term complications, even after 12 years of use.

Who Should Not Take UDCA?

Your doctor may advise you not to take UDCA if you:

Are allergic to bile salts
Have a bile duct blockage

What Is Elafibranor?

This medicine, sold under the brand name Iqirvo, is part of a class of drugs called PPAR agonists. It treats PBC by reducing the amount of bile in your body. It comes as a once-daily pill.

How effective is elafibranor?

In clinical trials, more than half of those taking part who took 80 milligrams of Iqirvo daily had a biochemical response, compared to just 4% of those taking a placebo, or inactive treatment.

What are the side effects of elafibranor?

The most common side effects people have had from taking Iqirvo are:

Weight gain or loss
Diarrhea
Belly pain
Nausea and vomiting
Joint pain
Constipation
Fractures
Gastroesophageal reflux disease (GERD)
Dry mouth
Rash

Is elafibranor safe?

Besides minor side effects, all medications have some risk of more serious complications. Some of these may mean that certain people should not take a specific drug.

This medicine isn't recommended for people with very severe cirrhosis and liver damage.

Elafibranor can raise your likelihood of having a bone fracture. Talk to your doctor about ways to protect your bones while you take this medicine.

Who should not take elafibranor?

You should not take elafibranor if you have advanced cirrhosis or a complete obstruction of your bile ducts.

What Is Obeticholic Acid (OCA)?

Obeticholic acid (OCA) is another bile salt. It’s in a drug class called farnesoid X receptor (FXR) agonists. FXR is a protein found in the liver and intestine that regulates your body’s production of bile, among other things.

This medicine helps your body make bile acids and move them through your liver. It may also reduce liver scarring and inflammation. OCA comes as a 5 milligram (mg) pill that you take once a day. Your doctor can bump up the dose to 10 mg after three months if you haven't improved enough on the lower dose.

How effective is OCA?

In studies, people who took OCA had improvements in tests of liver function and inflammation. Combining OCA with UDCA may lower the risk of cirrhosis, liver cancer, and death from liver disease, as well as reduce the need for a liver transplant.

What are the side effects of OCA?

The main side effect from OCA is itching, which is also a symptom of PBC. Other common side effects include:

Tiredness
Belly pain
Rash
Dizziness
Constipation

Is OCA safe?

Because this medicine can make liver function worse, it carries a boxed warning. This is a warning the FDA puts on medications that carry a serious risk of harmful effects. The warning on OCA is about a greater chance of liver injury for anyone with severe or advanced cirrhosis, or with high pressure in the portal vein that carries blood from organs like the stomach and intestines to the liver.

Who should not take OCA?

You should not take OCA if you have one of the conditions covered by the FDA boxed warning. Your doctor may advise you not to take OCA if you have a complete blockage of your bile ducts.

What Is Seladelpar?

Seladelpar (Livdelzi) is a newer treatment for PBC and is also a PPAR agonist. This type of drug activates a type of protein called a peroxisome proliferator activated receptor. When active, these proteins regulate how certain genes work. With PBC, they ease inflammation and block the formation of bile acids.

Livdelzi is a once-daily capsule that you take along with UDCA if UDCA alone didn't help you enough.

How effective is seladelpar?

In a clinical trial, 61.7% of those who took seladelpar had a clinical response, compared to 20% of those who did not. About 25% reached normal alkaline phosphatase levels, while none of the participants who did not take seladelpar had this result. About 94% of those taking part in the study received UDCA as standard treatment.

What are the side effects of seladelpar?

In studies, people who took Livdelzi for one year saw improvements in their liver function tests. After six months on this drug, itching improved. The most common side effects with this drug are:

Headaches
Belly pain
Nausea
Bloating
Dizziness

Is seladelpar safe?

Research has shown that people who took Livdelzi for up to three years reported no new serious symptoms and had no increase in side effects.

Who should not take seladelpar?

Your doctor may advise you not to take seladelpar if you:

Are allergic to seladelpar
Have advanced liver disease, including cirrhosis

Medications to Treat Your Symptoms

In addition to medications that help to improve liver function and slow the progression of PBC, your doctor may prescribe other drugs that help to ease some of the symptoms that come along with this condition:

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How Much Do PBC Medications Cost?

The price of a medication for PBC will depend on several things, including:

Which drug you take
The dosage
Insurance coverage
Your pharmacy
Your access to patient assistance programs (PAPs)

Without insurance coverage, rebates, or coupons, a 30-day supply of UDCA generally costs under $100. Elafibranor, OCA, and seladelpar can cost tens of thousands of dollars per 30-day cycle.

Many drug companies that make these medications offer PAPs to help reduce out-of-pocket costs. They may give you the medication at a reduced cost or, sometimes, for free. You may need to meet certain income standards to qualify.

You may also be able to get a copay card to reduce the out-of-pocket price. Some drug companies will also help answer questions you have about your insurance coverage or other ways to get financial help.

You can find information about financial assistance for PBC medications at these sources:

Clinical Trials of PBC Treatments

Researchers continue to do clinical trials to study how safe PBC medications are and how well they work.

You can visit clinicaltrials.gov to find studies across the country. For each trial listed, you’ll find information on:

The research goal
How the trial is set up
Whether it’s still recruiting
Where it's located
What conditions you must meet to qualify

If you're interested in taking part in a clinical trial, ask your doctor to suggest one that might be a good fit for you.

5 Things to Consider When Choosing a Medication Treatment for PBC

When deciding on a PBC medicine, talk to your doctor about your treatment goals and the benefits and risks of each drug. Here are five important things to ask about.

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Disease severity. Treatments aim to make PBC less severe. But if you already have severe cirrhosis and you have lost a lot of liver function, some of these medicines may not be safe for you to take.

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Past treatments. UDCA is usually the first treatment for PBC. If it doesn't improve your condition enough after one year, then your doctor might add another medicine, like OCA or Livdelzi.

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Other drugs you take. PBC drugs might react with other medicines you take, such as some antibiotics, cholesterol-lowering drugs, and antifungal medicines. Your doctor may recommend switching to a different medicine to avoid dangerous interactions.

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Symptoms. The main treatments for PBC help to slow the disease and prevent liver damage. Other medicines relieve symptoms like itching and fatigue. Some of these drugs can actually make itching worse, which could be something to think about when you and your doctor are choosing a treatment.

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Possible side effects. Each medicine comes with possible side effects. Some of the more common ones from PBC medicines are headaches, belly pain, nausea, and diarrhea. Ask your doctor which side effects you might have from the medicine they're prescribing, and how to manage those side effects if they happen to you.

Understanding Treatments for Primary Biliary Cholangitis (PBC)