What Is Primary Biliary Cholangitis? Symptoms, Diagnosis, and What's Changed in Treatment
By: Katie Wu, Emily Noronha, Claire Baum, Cait Williamson PhD
Posted: October 9, 2026
If you've been diagnosed with primary biliary cholangitis, or you're trying to understand a loved one's diagnosis, here's what it actually is, how doctors confirm it, and what the treatment journey looks like today.
What is primary biliary cholangitis?
PBC is a chronic autoimmune disease that affects the small bile ducts inside the liver. Bile ducts carry bile, a fluid your liver makes to help digest fat and clear out waste, from the liver into the intestine.
In PBC, the immune system mistakenly attacks the cells lining these small ducts. Over time, the ducts become inflamed and scarred, and bile backs up inside the liver instead of draining normally. That buildup is called cholestasis, and it's what damages liver tissue over the years, and, left untreated, can eventually progress to cirrhosis.
PBC used to be called "primary biliary cirrhosis," and you may still see that older name in some records or research. The name changed because most people diagnosed today are at an earlier stage and don't yet have cirrhosis. Catching PBC early, before scarring sets in, significantly affects long term outcomes.
Who gets PBC, and what are common symptoms?
PBC is considered a rare disease, and it disproportionately affects women, particularly those in their 40s to 60s. Researchers don't fully understand why the immune system targets the bile ducts, but genetics and environmental exposures both appear to play a role.
Some people with PBC have no symptoms in the early stages and are only diagnosed after a routine blood test turns up an abnormal liver enzyme called alkaline phosphatase, or ALP. When symptoms do appear, the two most common are:
- Fatigue that doesn't improve with rest and can be severe enough to affect daily life
- Itching (pruritus) which often starts on the hands and feet and can spread, sometimes worsening at night
Other symptoms can include dry eyes and mouth, discomfort in the upper right abdomen, and, in more advanced disease, jaundice (yellowing of the skin or eyes). Because fatigue and itching overlap with many other conditions, PBC is sometimes missed or misattributed for months or years before a diagnosis is made.
How is PBC diagnosed?
There's no single symptom that confirms PBC, so doctors typically look at three pieces of evidence together:
- Liver blood tests. An elevated ALP level, often alongside other liver enzymes, is usually the first clue.
- Antimitochondrial antibodies (AMA). Roughly 90 to 95 percent of people with PBC have this specific antibody in their blood, and its presence is considered highly specific to the disease. Some people with PBC are AMA-negative, so a negative result doesn't rule it out.
- Liver imaging or biopsy. Imaging can help confirm the pattern of bile duct damage, and in unclear cases, a liver biopsy provides a direct look at the tissue.
If you've had unexplained itching or fatigue along with abnormal liver tests, you may want to ask your doctor directly whether PBC has been considered and whether an AMA test has been run.
What's changed recently in PBC treatment
For decades, the foundation of PBC treatment has been a single drug: ursodeoxycholic acid, or UDCA. UDCA helps bile flow more easily and, in most patients, slows disease progression significantly. It remains the essential first-line therapy for everyone diagnosed with PBC.
The challenge is that UDCA doesn't work for everyone. Up to 40 percent of patients have an inadequate response, meaning ALP levels don't come down enough even with consistent use, and UDCA does little to relieve the itching and fatigue symptoms that many patients find most disruptive day to day.
For a long time, the main option for patients who don’t respond to UDCA was a drug called obeticholic acid (brand name Ocaliva). However in 2025, post-market data showed a risk of serious liver injury, including in patients without cirrhosis, and obeticholic acid was pulled from the U.S. market.
In its place, two newer medications have moved to the forefront of second-line PBC care:
- Elafibranor (brand name Iqirvo), which received FDA approval in 2024
- Seladelpar (brand name Livdelzi), also approved in 2024
Both belong to a class of drugs called PPAR agonists, which work through a different pathway than UDCA and obeticholic acid to reduce bile acid buildup and calm liver inflammation. In clinical trials, both drugs helped a meaningful share of patients bring ALP levels down, and importantly, both also showed improvement in pruritus, the itching that UDCA alone rarely helps. This is a fast-moving area of PBC care, and treatment decisions are highly individual.
What to bring up at your next appointment
If your last conversation with your medical provider about PBC treatment happened before 2024, your treatment options may have changed. That's true whether you're newly diagnosed, doing well on UDCA alone, or have been managing an inadequate response for years without feeling like your options were fully explored.
- "What is my current ALP level, and is it in the range considered an adequate response to treatment?"
- "If I was ever prescribed obeticholic acid, how has my treatment plan been updated since its withdrawal?"
- "Am I a candidate for elafibranor or seladelpar?"
Why patient registries matter for a disease like PBC
Because PBC is rare and its treatment landscape is changing quickly, much of what researchers know about how these newer therapies perform in the real world, beyond the controlled setting of a clinical trial, depends on data from patients actually living with the disease. Registries gather real-world experiences directly from patients so researchers can understand how treatments perform across different ages, disease stages, and backgrounds, not just within a trial's specific criteria. Registries also help researchers understand patient reported outcomes, or data that is shared in between doctor visits, to better understand different symptoms such as itching and fatigue.
If you're managing PBC, your unique experience is exactly the kind of information that helps build a fuller picture for the patients who come after you.
About Novellia: Novellia is a patient health platform that helps people consolidate their medical records, understand their health data, and participate in research studies on their own terms. Patients stay in control of their data at every step.
Sources
https://www.nejm.org/doi/full/10.1056/NEJMoa2306185
