The Food and Drug Administration has approved Livmarli™ (maralixibat) oral solution for the treatment of cholestatic pruritus in patients with Alagille syndrome 1 year of age and older.
Livmarli is a reversible inhibitor of the ileal bile acid transporter. It decreases the reabsorption of bile acids (primarily the salt forms) from the terminal ileum.
The approval was based on data from the phase 2b ICONIC study (ClinicalTrials.gov Identifier: NCT02160782), along with 5 years of data from supportive studies.
The ICONIC study evaluated the efficacy and safety of maralixibat 380mcg/kg once daily in patients 1 to 18 years of age with Alagille syndrome. Following 18 weeks of maralixibat treatment, patients were randomly assigned to receive either maralixibat (n=13) or placebo (n=16) for 4 weeks. After the randomized withdrawal period, all patients received maralixibat for an additional 26 weeks.
Treatment with maralixibat led to statistically significant reductions pruritus, as measured by the Itch Reported Outcome Instrument. On average, patients treated with maralixibat for 22 weeks maintained pruritus reduction, whereas those who were withdrawn from treatment after week 18 and received placebo returned to baseline pruritus scores by week 22. After re-entering the open-label treatment phase, both randomized treatment groups had similar mean pruritus scores by week 28.
The most common adverse reactions reported with maralixibat included diarrhea, abdominal pain, vomiting, fat-soluble vitamin deficiency, liver test abnormalities, gastrointestinal bleeding, and bone fractures.
Livmarli oral solution is supplied in a 30mL bottle; each mL contains 9.5mg of maralixibat.
- US 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. News release. Mirum Pharmaceuticals, Inc. September 29, 2021. Accessed September 30, 2021. https://apnews.com/hub/prescription-drugs.
- Livmarli. Package insert. Mirum Pharmaceuticals, Inc.; 2021. Accessed September 30, 2021. https://files.mirumpharma.com/livmarli/livmarli-prescribinginformation.pdf.