IMPROVEMENTS IN ITCH

Results with 
LIVMARLI

The goal of LIVMARLI is to provide lasting itch relief for patients with Alagille syndrome who are 3 months of age and older.

Once-daily LIVMARLI provides meaningful, long-lasting improvements in itchiness for patients with Alagille syndrome.

Will LIVMARLI work right away?

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LIVMARLI can work within weeks

For many patients taking LIVMARLI, itch relief was seen at the very first assessment.* Full improvement in the itch was felt by 3 to 4 months on LIVMARLI, and this relief was maintained through the first year.

*Itch relief was first assessed at Week 3.

How well does LIVMARLI work?

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Patients with Alagille syndrome were treated with LIVMARLI in a 4-year clinical study. During that time, many patients taking LIVMARLI experienced significantly less itchiness.

Pie chart displaying the percentage of patients who experienced less itchniness than they felt at the start of treatment

During the first year of treatment with once-daily LIVMARLI, more than 80% of patients experienced less itchiness than they felt at the start of treatment. This means that patients had an improvement in their Itch Reported Outcome (ItchRO) score of 1 or more.

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Patients who continued to take LIVMARLI after the first year (as part of the long-term part of the clinical study) experienced minimal to no itch for the vast majority of days on treatment.

What was the clinical study for LIVMARLI?

Patients were treated with LIVMARLI in a 4-year clinical study. The purpose of the study was to find out if LIVMARLI helped reduce a patient’s level of itch.

How did you monitor a patient’s itch?

In the clinical study, patients kept an ongoing log of their itch using the Itch Reported Outcome (ItchRO) tool. This tool was used to measure itch severity over time based on a 5-point scale:

0
Not itchy at all
1
A little bit itchy
2
Somewhat itchy
3
Very itchy
4
Extremely itchy

What was considered to be an improvement in itch?

Changes in ItchRO score by 1 point or more represent noticeable differences in the itch; for example, a change from 3 to 2 means that a patient went from feeling very itchy to feeling somewhat itchy.

What is the long-term impact of itch improvements?

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Some patients with Alagille syndrome continued taking LIVMARLI during 3 long-term clinical studies.

Patients who experienced a significant improvement in their itch were also more likely to experience transplant-free survival, meaning that they were alive and did not have a liver transplant.

For those who achieved more than a 1-point reduction in ItchRO(Obs), 93% were alive and did not have a transplant 6 years after starting LIVMARLI.

  • For those who did not achieve more than a 1-point reduction in ItchRO(Obs), only 57% remained alive and did not have a transplant 6 years after starting LIVMARLI

The impact of LIVMARLI medicine on transplant-free survival has not been established. No information about liver fibrosis (scarring) was collected.

How was the impact on the liver studied?

There were 3 clinical studies conducted for patients with Alagille syndrome. Some patients from those studies chose to stay on LIVMARLI long term. These patients were observed to see if there were any factors that could predict transplant-free survival.

Patients were included if they had moderate-to-severe itch, were considered to be benefitting from LIVMARLI, stayed on treatment for at least 48 weeks, and had lab results at 48 weeks. All patients took LIVMARLI.

What do you mean by “transplant-free survival”?

Transplant free means patients were still alive and had not undergone a liver transplant. Transplant-free survival refers to the percentage of patients who are alive and have not gotten a liver transplant at a certain point in time.

How did the studies work?

In the clinical studies, patients kept an ongoing log of their itch using the Itch Reported Outcome (ItchRO) tool. They were then assessed to determine the relationship between their itch intensity score and transplant-free survival.

More Info About ItchRO

Patients used this tool to measure itch severity over time based on a 5-point scale:

  • 0=Not itchy at all
  • 1=A little bit itchy
  • 2=Somewhat itchy
  • 3=Very itchy
  • 4=Extremely itchy

So, for those who had more than a 1-point improvement in their itch—for example, going from “very itchy” to “a little bit itchy”—93% were alive with their liver after 6 years of taking LIVMARLI.

3
Very itchy
1
A little bit itchy

For those who had a 1-point improvement in their itch or less—for example, going from "very itchy" to "somewhat itchy"—57% were alive with their liver after taking LIVMARLI for 6 years.

Get more information about the 
long-term impact of LIVMARLI

Lower Costs Within Reach

Lower Costs Within Reach

Mirum Access Plus works closely with your doctor and insurance plan to help facilitate coverage for LIVMARLI.

Talk to Your Doctor About LIVMARLI

Talk to Your Doctor About LIVMARLI

Get the conversation started about whether LIVMARLI is right for you or your child.

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Indication

LIVMARLI is a prescription medicine used to treat cholestatic pruritus (itch) in patients who are 3 months of age and older with Alagille syndrome.

It is not known if LIVMARLI is safe and effective in children with Alagille syndrome who are under 3 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?

  • Liver injury. Changes in certain liver tests are common in patients 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 and physical exams 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
    • 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 Alagille syndrome 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 if you have certain side effects.

LIVMARLI is taken by mouth, 1 time each day, 30 minutes before a meal in the morning. 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.