What is Alagille syndrome?

Alagille syndrome is a rare, multisystem disease that largely affects the liver. It can also affect 
other major parts of the body, including the heart, face, eyes, kidneys, and spine.


What causes Alagille syndrome?

The liver makes a digestive fluid called bile that flows from the liver to the gallbladder and small intestine through a network of tubes called bile ducts. Bile contains bile acids, which help with the digestion and absorption of fats.

Bile acid buildup in Alagille syndrome

Bile acid buildup

For people with Alagille syndrome, bile cannot properly drain out of the liver. This results in a buildup of bile acids in the liver and blood.

Research suggests that this buildup of bile acids in the liver may be related to a common symptom: intense itch, also known as cholestatic pruritus.

The itch in Alagille syndrome

Itchiness is often a very challenging symptom in Alagille syndrome. Some people scratch so hard and so frequently that it causes red marks, bleeding, and scarring.

Intense itchiness has also been reported to 
be a leading reason for liver transplant.

Liver transplant
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What else should you know about Alagille syndrome?


Symptoms start early.

Signs and symptoms of Alagille syndrome often appear in infancy or early childhood.

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Only scratching the surface.

Until now, ways to manage the itch associated with Alagille syndrome have been limited. Historically, the available medicines were not made specifically for Alagille syndrome. For very severe cases, the other choices may be surgery or a liver transplant.

But LIVMARLI may be able to help.

LIVMARLI is the first and only FDA-approved treatment for the itch in patients with Alagille syndrome who are 1 year of age and older.


Understanding LIVMARLI

What it is. Who it’s for. How it works.

Learn About LIVMARLI
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LIVMARLI is a prescription medicine used to treat cholestatic pruritus (itch) in patients with Alagille syndrome 1 year of age and older. It is not known if LIVMARLI is safe and effective in children under 1 year of age or in adults 65 years and older.

Important Safety Information

What are the possible side effects 

LIVMARLI can cause serious side effects, including:

Changes in liver tests. Changes in certain liver tests are common in patients with Alagille syndrome but may worsen during treatment with LIVMARLI. These changes may be a sign of liver injury and can be serious. Your health care provider should do blood tests before starting and during treatment to check your liver function. Tell your health care provider right away if you get any signs or symptoms of liver problems, including:

  • nausea or vomiting
  • skin or the white part of the eye turns yellow
  • dark or brown urine
  • pain on the right side of the stomach (abdomen)
  • loss of appetite

Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea, stomach pain, and vomiting during treatment. Tell your health care provider right away if you have any of these symptoms more often or more severely than normal for you.

A condition called Fat-Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamins A, D, E, and K) stored in body fat. FSV deficiency is common in patients with Alagille syndrome but may worsen during treatment. Your health care provider should do blood tests before starting and during treatment.

Other common side effects reported during treatment were bone fractures and gastrointestinal bleeding.

Tell your health care provider about all medicines that you take. LIVMARLI may affect the way some other medicines work, and some other medicines may affect the way LIVMARLI works. If you take a medicine that lowers cholesterol by binding bile acids, such as cholestyramine, colesevelam, or colestipol, take it at least 4 hours before or 4 hours after you take LIVMARLI.

LIVMARLI is taken by mouth, 1 time each day, 30 minutes before the first meal of the day. Be sure to use the provided oral dosing dispenser to accurately measure the dose of medicine.

These are not all of the possible side effects of LIVMARLI. Call your health care provider for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

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