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  Indication
Important Safety Information
Prescribing Information

What Is Progressive Familial 
Intrahepatic Cholestasis (PFIC)?

PFIC is a group of rare, inherited diseases that mostly affects the liver. There are 3 main types of PFIC and several other, rarer types. These are all called subtypes. The 2 most common subtypes are PFIC2 and PFIC3.

The 3 main types of PFIC

PFIC1 is caused by changes to a protein in the liver called familial intrahepatic cholestasis associated protein 1 (FIC1). PFIC1 is not very common.
PFIC2 refers to changes to a protein in the liver called the bile salt export pump (BSEP). PFIC2 is the most common type and makes up approximately half of all cases.
PFIC3 results from changes to a protein in the liver called multidrug resistance class III (MDR3). PFIC3 makes up approximately one-third of all cases.

How does PFIC affect the liver?

The liver makes a fluid called bile that helps with the digestion of fats. Bile acids, which are a part of bile, are made by the liver cells.

In PFIC, bile acids get stuck inside the cells. 
Over time, bile acids build up in the liver and blood.
Mechanism of Disease Infographic
95% of bile acids made by the liver are recycled in the intestine through the IBAT.

What are the effects of bile acid buildup?

When bile acids build up in the liver, they spill over into the bloodstream. This can cause an increase in bile acids throughout the entire body. These high levels of bile acids are an underlying cause of cholestatic pruritus (itch) in PFIC.

A COMMON AND CHALLENGING SYMPTOM

Cholestatic pruritus affects most people with PFIC (76% to 100%) and is also the most burdensome symptom.

The scratching is a signal

Over time, the buildup of bile acids in the liver can lead to long-term problems, including:

Inflammation
Damage to the liver that worsens over time
Scar tissue in the liver (fibrosis)
Bile acid buildup causes immediate problems (cholestatic pruritus) and has long-term impacts (liver problems).

Research has shown that approximately

50%

of patients with PFIC1 or PFIC2 still have their own liver by age 10.

50 Percent Native Liver Survival Chart
  • In PFIC2, specifically, only 32% of patients still have their own liver by age 18
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Cholestatic pruritus is more than just scratching

Cholestatic pruritus is often a very challenging symptom for people with PFIC.

Cholestatic pruritus is frequently first seen by age 3 months in people with PFIC1 and PFIC2 and by age 2 years to 3 years in people with PFIC3.

Beyond the scratching, it can be difficult to navigate all aspects of cholestatic pruritus without the right support. Signs and symptoms related to cholestatic pruritus can look different during the day and/or night and vary for each person.

The burden of cholestatic pruritus

Approximately 60% of caregivers report that their child suffers from sleep disturbances due to itch.

Sleep disturbances include needing help falling asleep, needing soothing, and sleeping with a caregiver.

Additional signs and symptoms related to cholestatic pruritus can include:

Skin damage
Worsened performance in school
Negative impact on social activities
Irritability
Worsened physical function
Worsened general health
Cognitive impact
Burning, tingling, or prickling sensation

ALL LEVELS OF CHOLESTATIC PRURITUS DESERVE ATTENTION

Share your experience with cholestatic pruritus with your doctor and let them know about any changes in symptoms. Not sure where to start? Visit our Resources page before your next doctor’s appointment to help guide the discussion.

Talking to Your Doctor

What else should you know about cholestatic pruritus in PFIC?

Symptoms
start early

Cholestatic pruritus is frequently first seen by age 3 months in people with PFIC1 and PFIC2 and by age 2 years to 3 years in people with PFIC3.

Only scratching
the surface

Treatment options for cholestatic pruritus in PFIC have been limited. In cases where cholestatic pruritus is unmanageable, many people still choose to have surgery and/or a liver transplant.

Cholestatic pruritus
is often normalized and overlooked

It can be hard to recognize the impact of cholestatic pruritus on daily life. Symptoms of cholestatic pruritus may start to seem normal or become routine over time, making them easier to downplay or minimize.

LIVMARLI logo

Understanding LIVMARLI

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

Learn About LIVMARLI

INDICATION

LIVMARLI is a prescription medicine used to treat cholestatic pruritus (itch) in patients 12 months of age and older with progressive familial intrahepatic cholestasis (PFIC).

LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein.

It is not known if LIVMARLI is safe and effective in children with PFIC who are under 12 months of age. It is not known if LIVMARLI is safe and effective in adults who are 65 years of age and older.

IMPORTANT SAFETY INFORMATION

What are the possible side effects 
of LIVMARLI?

LIVMARLI can cause serious side effects, including:

  • Liver injury. Changes in certain liver tests are common in patients with PFIC but may worsen during treatment. These changes may be a sign of liver injury and can be serious or may lead to transplant or death. Your health care provider should do blood tests and physical exams before starting and during treatment with LIVMARLI 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
    • your skin or the white part of your eye turns yellow
    • dark or brown urine
    • pain on the right side of your stomach (abdomen)
    • fullness, bloating, or fluid in your stomach area (ascites)
    • loss of appetite
    • bleeding or bruising more easily than normal, including vomiting blood
  • Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain during treatment. Diarrhea can also cause the loss of too much body fluid (severe dehydration). Your health care provider may advise you to monitor for new or worsening stomach problems, including stomach pain, diarrhea, blood in your stool, or vomiting

    Tell your health care provider right away if you have any new or worsening signs or symptoms of stomach and intestinal problems, including:
    • diarrhea
    • more frequent bowel movements than usual
    • stools that are black, tarry, or sticky, or have blood or mucous
    • severe stomach-area pain or tenderness
    • vomiting
    • urinating less often than usual
    • dizziness
    • headache
  • A condition called Fat-Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat. FSV deficiency is common in patients with PFIC but may worsen during treatment. Your health care provider should do blood tests before starting and during treatment, and may monitor for bone fractures and bleeding, which are common side effects

Tell your health care provider about all medicines that you take, as LIVMARLI may interact with other medicines. 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.

Your health care provider may change your dose, or temporarily or permanently stop treatment with LIVMARLI if you have certain side effects.

LIVMARLI is taken by mouth, 2 times each day, 30 minutes before a meal. 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. For more information, ask your health care provider or pharmacist. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.

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INDICATION

LIVMARLI is a prescription medicine used to treat cholestatic pruritus (itch) in patients 12 months of age and older with progressive familial intrahepatic cholestasis (PFIC).

LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein.

It is not known if LIVMARLI is safe and effective in children with PFIC who are under 12 months of age. It is not known if LIVMARLI is safe and effective in adults who are 65 years of age and older.

IMPORTANT SAFETY INFORMATION

What are the possible side effects 
of LIVMARLI?

LIVMARLI can cause serious side effects, including:

  • Liver injury. Changes in certain liver tests are common in patients with PFIC but may worsen during treatment. These changes may be a sign of liver injury and can be serious or may lead to transplant or death. Your health care provider should do blood tests and physical exams before starting and during treatment with LIVMARLI 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
    • your skin or the white part of your eye turns yellow
    • dark or brown urine
    • pain on the right side of your stomach (abdomen)
    • fullness, bloating, or fluid in your stomach area (ascites)
    • loss of appetite
    • bleeding or bruising more easily than normal, including vomiting blood
  • Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain during treatment. Diarrhea can also cause the loss of too much body fluid (severe dehydration). Your health care provider may advise you to monitor for new or worsening stomach problems, including stomach pain, diarrhea, blood in your stool, or vomiting

    Tell your health care provider right away if you have any new or worsening signs or symptoms of stomach and intestinal problems, including:
    • diarrhea
    • more frequent bowel movements than usual
    • stools that are black, tarry, or sticky, or have blood or mucous
    • severe stomach-area pain or tenderness
    • vomiting
    • urinating less often than usual
    • dizziness
    • headache
  • A condition called Fat-Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat. FSV deficiency is common in patients with PFIC but may worsen during treatment. Your health care provider should do blood tests before starting and during treatment, and may monitor for bone fractures and bleeding, which are common side effects

Tell your health care provider about all medicines that you take, as LIVMARLI may interact with other medicines. 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.

Your health care provider may change your dose, or temporarily or permanently stop treatment with LIVMARLI if you have certain side effects.

LIVMARLI is taken by mouth, 2 times each day, 30 minutes before a meal. 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. For more information, ask your health care provider or pharmacist. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.