What is Obeticholic Acid: Uses, Dosage, Side Effects and More

Obeticholic acid is a prescription medication that helps treat certain liver diseases by mimicking a natural substance your body makes to process bile. This medication works specifically for people with primary biliary cholangitis, a rare autoimmune condition that slowly damages the bile ducts in your liver.

If your doctor has prescribed this medication, you likely have questions about how it works and what to expect. Let's walk through everything you need to know about obeticholic acid in simple, straightforward terms.

What is Obeticholic Acid?

Obeticholic acid is a synthetic version of a bile acid that naturally occurs in your body. It belongs to a class of medications called farnesoid X receptor agonists, which sounds complicated but simply means it activates specific receptors in your liver.

Your liver normally produces bile acids to help digest fats and remove waste products. When you have certain liver conditions, this process doesn't work properly. Obeticholic acid steps in to help restore some of this normal function by signaling your liver to reduce bile acid production and decrease inflammation.

This medication is relatively new to the market, having been approved by the FDA in 2016. It represents an important advancement in treating rare liver diseases that previously had limited treatment options.

What is Obeticholic Acid Used For?

Obeticholic acid is primarily prescribed to treat primary biliary cholangitis (PBC), formerly known as primary biliary cirrhosis. This is a chronic autoimmune disease where your immune system mistakenly attacks the small bile ducts in your liver.

Your doctor might prescribe this medication if you have PBC and either can't tolerate ursodeoxycholic acid (the first-line treatment) or haven't responded well to it. The goal is to slow down the progression of liver damage and reduce the risk of complications like cirrhosis.

Currently, obeticholic acid is specifically approved for adults with PBC. Researchers are studying its potential use for other liver conditions, including non-alcoholic steatohepatitis (NASH), but these uses are still experimental.

How Does Obeticholic Acid Work?

Obeticholic acid works by activating farnesoid X receptors in your liver, intestines, and kidneys. Think of these receptors as switches that control how your body handles bile acids and inflammation.

When you take this medication, it tells your liver to reduce the production of bile acids and decreases the uptake of bile acids from your intestines. This helps reduce the toxic buildup of bile acids that can damage liver cells in people with PBC.

The medication also has anti-inflammatory effects, which can help slow down the immune system's attack on your bile ducts. This is considered a moderately strong medication that can significantly impact liver function tests, but it works gradually over months rather than providing immediate relief.

How Should I Take Obeticholic Acid?

You should take obeticholic acid exactly as your doctor prescribes, typically once daily with or without food. The medication comes as tablets that you swallow whole with water.

Most people start with a lower dose that may be increased gradually based on how well you tolerate the medication and how your liver responds. Your doctor will monitor your liver function tests regularly to determine the right dose for you.

You can take this medication with or without food, but try to take it at the same time each day to maintain consistent levels in your body. If you have trouble swallowing pills, talk to your doctor about your options, but don't crush or break the tablets.

How Long Should I Take Obeticholic Acid For?

Obeticholic acid is typically a long-term treatment that you'll need to continue indefinitely to maintain its benefits. Since PBC is a chronic condition, stopping the medication usually means the disease will continue to progress.

Your doctor will regularly monitor your response to the medication through blood tests that check your liver function. These tests help determine whether the medication is working effectively and if any dose adjustments are needed.

The length of treatment varies for each person, but many people with PBC need to take liver medications for life. Your doctor will discuss your individual treatment plan and help you understand what to expect for your specific situation.

What Are the Side Effects of Obeticholic Acid?

Like all medications, obeticholic acid can cause side effects, though not everyone experiences them. The most common side effect is itching, which affects many people taking this medication.

Here are the more common side effects you might experience:

• Severe itching (pruritus), especially at night
• Fatigue or feeling unusually tired
• Abdominal pain or discomfort
• Joint pain
• Throat pain
• Dizziness
• Constipation
• Skin rash

The itching can be quite bothersome and may interfere with sleep. If this happens, your doctor might adjust your dose or recommend treatments to help manage the itching.

Less common but more serious side effects can include:

• Worsening liver function (rare but possible)
• Severe allergic reactions
• Significant changes in cholesterol levels
• Gallbladder problems

Contact your doctor immediately if you experience severe abdominal pain, signs of liver problems like yellowing of your skin or eyes, or any severe allergic reactions.

Who Should Not Take Obeticholic Acid?

Obeticholic acid isn't suitable for everyone, and your doctor will carefully evaluate whether it's right for you. People with certain conditions should avoid this medication or use it with extreme caution.

You should not take obeticholic acid if you have:

• Complete biliary obstruction (blocked bile ducts)
• Severe liver cirrhosis (Child-Pugh Class B or C)
• Known allergy to obeticholic acid or any of its ingredients
• Severe kidney disease

Your doctor will also be cautious about prescribing this medication if you're pregnant, breastfeeding, or planning to become pregnant, as there isn't enough safety data for these situations.

People with a history of gallbladder disease, high cholesterol, or other liver conditions may need special monitoring while taking this medication. Always tell your doctor about all your medical conditions and medications before starting treatment.

Obeticholic Acid Brand Names

The brand name for obeticholic acid is Ocaliva, manufactured by Intercept Pharmaceuticals. This is currently the only brand available in the United States.

Ocaliva comes in tablet form in different strengths, typically 5 mg and 10 mg tablets. Your doctor will prescribe the appropriate strength based on your individual needs and response to treatment.

Generic versions of obeticholic acid are not yet available in the United States, so Ocaliva is the only option currently. This medication can be quite expensive, so discuss insurance coverage and patient assistance programs with your doctor and pharmacist.

Obeticholic Acid Alternatives

If you can't take obeticholic acid or it doesn't work well for you, there are other treatment options for PBC. The most common alternative is ursodeoxycholic acid (UDCA), which is often the first-line treatment.

Other alternatives your doctor might consider include:

• Ursodeoxycholic acid (Actigall, Urso) - often tried first
• Bezafibrate (not FDA-approved for PBC but used off-label)
• Budesonide (for certain cases)
• Liver transplantation (for advanced cases)

Your doctor will work with you to find the best treatment approach based on your specific situation, other health conditions, and how well you tolerate different medications. Sometimes a combination of treatments works better than a single medication.

Is Obeticholic Acid Better Than Ursodeoxycholic Acid?

Obeticholic acid and ursodeoxycholic acid (UDCA) work differently and serve different roles in treating PBC. UDCA is typically the first medication doctors try because it has been used safely for many years.

Obeticholic acid is generally reserved for people who don't respond adequately to UDCA or can't tolerate it. Studies show that obeticholic acid can be more effective than UDCA alone in improving certain liver function tests.

However, "better" depends on your individual situation. UDCA tends to have fewer side effects and has been used safely for decades. Obeticholic acid may be more potent but can cause more bothersome side effects like severe itching.

Your doctor will consider your specific lab results, symptoms, and how well you've responded to other treatments when deciding which medication is best for you. Sometimes the two medications are used together for enhanced effectiveness.