The goal of LIVMARLI is to provide lasting itch relief for patients with Alagille syndrome who are 3 months of age and older.
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.
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.
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.
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.
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.
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:
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.
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.†
†The impact of LIVMARLI medicine on transplant-free survival has not been established. No information about liver fibrosis (scarring) was collected.
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.
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.
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.
Patients used this tool to measure itch severity over time based on a 5-point scale:
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.
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.
Mirum Access Plus works closely with your doctor and insurance plan to help facilitate coverage for LIVMARLI.
Get the conversation started about whether LIVMARLI is right for you or your child.
LIVMARLI is a prescription medicine used to treat cholestatic pruritus (itch) in patients with Alagille syndrome 3 months of age and older. It is not known if LIVMARLI is safe and effective in children under 3 months of age or in adults 65 years and older.
What are the possible side effects
of LIVMARLI?
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:
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 gastrointestinal bleeding and bone fractures.
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 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. Call your health care provider for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.