Livmarli (maralixibat)
An approved treatment for Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis.
Drug facts
The same compound appears under different names depending on the context. Here is how to identify Maralixibat wherever you encounter it, plus the key facts at a glance.
- Generic name
- Maralixibat
- Brand name
- Livmarli
- Development codes
- SHP625, LUM001
- Drug class
- Ileal bile acid transporter (IBAT) inhibitor
- Manufacturer
- Mirum Pharmaceuticals
- How it's taken
- Taken by mouth 30 minutes before a meal.
A liquid or tablet taken by mouth that treats the severe itch of cholestatic liver disease. It is approved for itch in Alagille syndrome from 3 months of age and in PFIC from 12 months, and it was the first drug approved for Alagille syndrome.
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Where Maralixibat fits
One of 2 FDA-approved IBAT inhibitors for itch in Alagille syndrome and PFIC, alongside Bylvay (odevixibat). Livmarli is approved for Alagille syndrome from 3 months and PFIC from 12 months, while Bylvay covers PFIC from 3 months and Alagille syndrome from 12 months.
How Maralixibat works
In Alagille syndrome and PFIC, bile does not drain properly from the liver, and bile acids build up in the body. That buildup causes an intense itch that can disrupt sleep, school and family life. Normally about 95% of bile acids are reabsorbed in the last part of the small intestine and sent back to the liver. Maralixibat blocks the pump that does this, so more bile acids leave in the stool and less builds up. It treats the itch and does not cure the underlying liver disease.
Side effects and safety
- Liver injury. Liver tests before starting and often during the first 6-8 months. Livmarli is stopped for good after repeated liver test problems, hepatitis that returns on restarting or signs of liver failure.
- Diarrhea and stomach pain. The dose is lowered or paused for ongoing diarrhea, blood in the stool, vomiting, dehydration or fever.
- Low vitamins A, D, E and K. Levels are checked before and during treatment. Fractures and bleeding were more common than on placebo, and treatment may pause if they happen.
- Propylene glycol in the liquid. Propylene glycol can be harmful in large amounts to children under 5, so the doctor counts it in the child's total daily intake.
- Liver tests at baseline and often in the first 6-8 months
- Vitamins A, D, E and K before and during treatment
- Watch for diarrhea, blood in the stool and signs of dehydration
Livmarli has no boxed warning, but it must not be used by people who have had liver decompensation. That includes bleeding from swollen veins, fluid in the belly or confusion from liver failure. Its warnings cover liver injury, diarrhea and stomach pain, and low levels of vitamins A, D, E and K. The liquid also contains propylene glycol, which matters for children under 5.
In the PFIC trial and its extension, 4 people had drug-related liver injury, and 1 needed a transplant and 1 died. In the PFIC trial, diarrhea affected 57% on Livmarli against 20% on placebo.
This is not a complete list of side effects. Talk to your doctor or pharmacist about what to expect and when to seek medical attention.
Taking Maralixibat
Taken by mouth 30 minutes before a meal. For Alagille syndrome it is taken once a day in the morning, starting at 190mcg/kg and rising after 1 week to 380mcg/kg. The daily maximum is 28.5mg as liquid or 30mg as tablets. For PFIC it builds up to 570mcg/kg twice a day, up to 38mg a day as liquid or 40mg as tablets. The 2 liquid strengths are not interchangeable for PFIC.
Tablets of 10mg, 15mg, 20mg and 30mg are for people who weigh at least 25kg and can swallow them. Bile acid binders such as cholestyramine are taken at least 4 hours apart from Livmarli.
Availability and cost
Only available as the brand-name product.
Help paying for Livmarli
Pick your insurance to see which help fits. Drugmaker copay cards can't be used with Medicare, Medicaid or TRICARE; charity funds are the usual route there.
- Insurance and case manager help
A Patient Navigator checks insurance benefits, helps if coverage is denied and arranges home delivery from the program's pharmacy.
The official page does not say who qualifies. Ask the program. · source - Copay help
Savings program for eligible commercially or privately insured patients, who may pay as little as $0 per fill, subject to program terms.
For: private insurance · source - Free medicine program
Patient Assistance Program for people who meet financial criteria.
For: no insurance, underinsured · source
Good to know: Call 1-855-MRM-4YOU (1-855-676-4968), weekdays 8am-8pm ET. Mirum's pages do not publish the full copay terms; ask the Patient Navigator about Medicare and other government coverage.
- From a charity · Alagille Syndrome AllianceALGSAssistance Program fundOpen
Pays for: Rent or mortgage, utilities, insurance premiums, counseling, and medical, adaptive or mobility equipment, up to $1,000 per year.
The foundation says: “Apply now (reviewed case by case as funding allows)” - From a charity · HealthWell FoundationAlagille Syndrome Vitamins and Supplements fundOpen
Pays for: Copays, premiums or other treatment costs.
- From a charity · PFIC NetworkPFIC Financial Assistance Program fundOpen
Pays for: Uncovered medical expenses (genetic testing, vitamins, travel), insurance premiums and copays, rent or utilities, up to $1,000 per year.
The foundation says: “The PFIC Network accepts applications throughout the year on a rolling basis.”
Every program and charity fund for Alagille Syndrome →
Every program and charity fund for Progressive Familial Intrahepatic Cholestasis →
Access and eligibility
Approved for cholestatic itch in Alagille syndrome from 3 months of age and in PFIC from 12 months. It is not recommended for a subgroup of PFIC type 2 with ABCB11 changes that leave no working bile salt export pump (BSEP) protein. It must not be used after liver decompensation, and its safety at 65 and older is not known.
Source: Mirum Access Plus (Livmarli patient support)
Access program details are provided for informational purposes and may vary based on insurance coverage, geographic location, and individual circumstances. Confirm current eligibility directly with the manufacturer or your specialty pharmacy.
Clinical trial results
For Alagille syndrome, the ICONIC trial (NCT02160782) gave Livmarli to 31 children, then randomly switched 29 of them to Livmarli or placebo for 4 weeks. Itch scores stayed low on Livmarli and rose on placebo, ending at 1.6 against 3.0 on a 0-4 scale. For PFIC, the MARCH-PFIC trial (NCT03905330) compared Livmarli with placebo for 26 weeks in 64 people with several PFIC types.
Morning itch scores fell 1.8 points on Livmarli against 0.6 on placebo, with less than a 1 in 10,000 chance of being a fluke. The 5 people with the most severe BSEP defect did not improve.
Development history
Mirum Pharmaceuticals won FDA approval on September 29, 2021 for itch in Alagille syndrome from age 1, the first drug for the condition. The age dropped to 3 months on March 13, 2023. PFIC was added from age 5 on March 13, 2024, then lowered to 12 months on July 24, 2024 with a stronger 19mg/mL liquid. The FDA approved Livmarli tablets on April 10, 2025.
Explore Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis trials
Other Alagille Syndrome & Progressive Familial Intrahepatic Cholestasis treatments
Cholestatic itch in Alagille syndrome and PFIC has 2 FDA-approved IBAT inhibitors: Livmarli (maralixibat), first approved September 29, 2021, and Bylvay (odevixibat), first approved July 20, 2021. Other care includes ursodiol, antihistamines and rifampin, used off-label for itch, plus fat-soluble vitamin supplements. Surgery to divert bile and liver transplant remain options when itch or liver disease cannot be controlled.
Common questions about Maralixibat
▸What is Maralixibat (Livmarli)?
A liquid or tablet taken by mouth that treats the severe itch of cholestatic liver disease. It is approved for itch in Alagille syndrome from 3 months of age and in PFIC from 12 months, and it was the first drug approved for Alagille syndrome.
▸How does Maralixibat work?
In Alagille syndrome and PFIC, bile does not drain properly from the liver, and bile acids build up in the body. That buildup causes an intense itch that can disrupt sleep, school and family life. Normally about 95% of bile acids are reabsorbed in the last part of the small intestine and sent back to the liver. Maralixibat blocks the pump that does this, so more bile acids leave in the stool and less builds up. It treats the itch and does not cure the underlying liver disease.
▸What are the side effects of Maralixibat?
Livmarli has no boxed warning, but it must not be used by people who have had liver decompensation. That includes bleeding from swollen veins, fluid in the belly or confusion from liver failure. Its warnings cover liver injury, diarrhea and stomach pain, and low levels of vitamins A, D, E and K. The liquid also contains propylene glycol, which matters for children under 5. In the PFIC trial and its extension, 4 people had drug-related liver injury, and 1 needed a transplant and 1 died. In the PFIC trial, diarrhea affected 57% on Livmarli against 20% on placebo.
▸How is Maralixibat taken?
Taken by mouth 30 minutes before a meal. For Alagille syndrome it is taken once a day in the morning, starting at 190mcg/kg and rising after 1 week to 380mcg/kg. The daily maximum is 28.5mg as liquid or 30mg as tablets. For PFIC it builds up to 570mcg/kg twice a day, up to 38mg a day as liquid or 40mg as tablets. The 2 liquid strengths are not interchangeable for PFIC. Tablets of 10mg, 15mg, 20mg and 30mg are for people who weigh at least 25kg and can swallow them. Bile acid binders such as cholestyramine are taken at least 4 hours apart from Livmarli.
▸Is Maralixibat FDA approved?
Yes, Maralixibat (Livmarli) is FDA approved (2021) for the treatment of Alagille Syndrome and Progressive Familial Intrahepatic Cholestasis.
▸What is the difference between Livmarli and Bylvay?
Both block the same bile acid pump in the gut to ease cholestatic itch. Livmarli is approved for Alagille syndrome from 3 months and PFIC from 12 months, while Bylvay covers PFIC from 3 months and Alagille syndrome from 12 months. Livmarli comes as a liquid or tablets taken before meals, and Bylvay as pellets or capsules taken with a meal.
▸Does Livmarli work for every type of PFIC?
It is not recommended for a subgroup of PFIC type 2 where ABCB11 changes leave no working BSEP protein. The 5 people with that severe defect in the main trial did not improve. Genetic test results help your child's liver doctor decide.
▸Why does my child need vitamin tests on Livmarli?
Low vitamins A, D, E and K are common in these liver diseases and can get worse on Livmarli. Low vitamin D and K can lead to fractures and bleeding, so levels are checked before and during treatment.
Sources and references
Every factual claim on this page is drawn from the public sources listed below. Click any reference to open the original document.
- U.S. National Library of Medicine, DailyMed · 2025-04-10. LIVMARLI (maralixibat) oral solution and tablets: prescribing information. https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=64000394-1ef6-4e76-8ba8-11f25ba1b167
- U.S. Food and Drug Administration · 2021-09-29. NDA 214662 approval letter. https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2021/214662Orig1s000Corrected_ltr.pdf
- U.S. Food and Drug Administration · 2024-07-24. NDA 214662 supplements S-009 and S-010 approval letter (PFIC from 12 months). https://www.accessdata.fda.gov/drugsatfda_docs/appletter/2024/214662Orig1s009,s010ltr.pdf
- Mirum Pharmaceuticals · 2025-04-14. Mirum's LIVMARLI Now FDA Approved in Tablet Formulation. https://ir.mirumpharma.com/news/news-details/2025/Mirums-LIVMARLI-Now-FDA-Approved-in-Tablet-Formulation/default.aspx
- ClinicalTrials.gov. Safety and Efficacy Study of LUM001 (Maralixibat) With a Drug Withdrawal Period in Participants With Alagille Syndrome (ALGS). https://clinicaltrials.gov/study/NCT02160782
- ClinicalTrials.gov. A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC). https://clinicaltrials.gov/study/NCT03905330
- Mirum Pharmaceuticals. LIVMARLI patient support. https://mirumaccessplus.com/livmarli-patient-support