What is Livmarli (maralixibat) for?

Livmarli (maralixibat) is an ileal bile acid transporter (IBAT) inhibitor indicated for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) aged 1 year and older.

It is available as oral solution, containing 9.5 mg/30 ml maralixibat.^[1]

How does Livmarli (maralixibat) work?

ALGS is a rare genetic condition caused by bile duct abnormalities. This causes a buildup of bile acids in the liver (cholestasis), which leads to inflammation and liver injury. This accumulation prevents the liver from working properly to eliminate waste from the bloodstream. Patients with cholestatic liver disease typically experience severe itching (pruritus), although it is not exactly understood how it develops.2

The active ingredient in Livmarli, maralixibat, is a reversible inhibitor of the ileal bile acid transporter (IBAT). It increases bile acid excretion in the fecus, thereby reducing the build-up of bile acids in the blood and liver that can cause itching and liver damage.2

Where has Livmarli (maralixibat) been approved?

Livmarli (maralixibat) was approved to treat cholestatic pruritus associated with ALGS by:

• The Food and Drug Administration (FDA), USA on September 29, 2021.^[3]

Together with the approval, the manufacturer received a rare pediatric disease priority review voucher.^[3]

Please note that this medicine may have also been approved in other regions than the ones we’ve listed. If you have a question about its approval in a specific country feel free to contact our support team.

How is Livmarli (maralixibat) taken?

The standard dosage is:^[1]

• The starting dose: 190 mcg/kg body weight taken by mouth (orally) once daily, taken 30 minutes before your first meal of the day.
• After one week: increase to 380 mcg/kg orally once daily, as tolerated.

The maximum daily dose volume for patients above 70 kg is 3 mL or 28.5 mg per day.^[1]

If you miss a dose of Livmarli, you should take it as soon as possible within 12 hours of the time it is usually taken. Then take your next dose at the usual time. If you miss a dose by more than 12 hours, do not take the missed dose. Take your next dose at the usual time.^[1]

If you take certain medicines for high cholesterol, such as cholestyramine, colesevelam, or colestipol, take it at least 4 hours before or 4 hours after you take Livmarli. Ask your treating doctor if you are not sure if you take these medicines.^[1]

The dose of Livmarli may have to be modified or (temporarily) stopped when your liver tests are abnormal.^[1]

Complete information about Livmarli (maralixibat) dosage (modifications) and administration can be found in the official prescribing information listed in our references section.^[1]

Note: Please consult with your treating doctor for personalised dosing.

Are there any known adverse reactions or side effects of Livmarli (maralixibat)?

Common adverse reactions

The most common side effects (≥5% of patients) listed in the prescribing information include:^[1]

• Diarrhea
• Belly (abdominal pain)
• Vomiting
• Fat-soluble vitamin deficiency
• Changes in liver tests
• Stomach and intestinal (gastrointestinal) problems, including gastrointestinal bleeding
• Bone fractures

Serious adverse reactions

The serious adverse effects listed in the prescribing information include:^[1]

• Serious liver injury

Use in a specific population

It is not known if Livmarli (maralixibat) will harm your unborn baby or if it passes into your breast milk. Talk to your treating doctor if you are pregnant or planning to become pregnant, or about the best way to feed your baby while taking this medicine.^[1]

For a comprehensive list of side effects and adverse reactions please refer to the official prescribing information.^[1]

References

1. Full prescribing information [FDA]: Livmarli (maralixibat) [PDF]
Mirum Pharmaceuticals, Sep 2021

2. IBAT inhibition
Mirum, cited Oct 2021

3. U.S. FDA Approves LIVMARLI (maralixibat) as the First and Only Approved Medication for the Treatment of Cholestatic Pruritus in Patients with Alagille Syndrome One Year of Age and Older
Mirum press release, Sep 2021