FDA Approves Livmarli for Treatment of Alagille Syndrome for Those 1 Year and Older


The agency has given the greenlight to maralixibat after the ICONIC study combined with 5 years of supportive studies that demonstrated the reduction of pruritis.

The FDA has approved Livmarli (maralixibat, Mirum), an oral solution for the treatment of cholestatic pruritus in individuals with Alagille syndrome (ALGS) who are aged 1 year and older, Mirum Pharmaceuticals, Inc, said in a statement.

“Children with Alagille syndrome suffer from cholestatic pruritus, which is serious, unremitting, and debilitating. Their sleep is disrupted, and they endure bleeding and scarring of the skin due to unrelenting scratching,” Binita Kamath, MBBChir, pediatric hepatologist at the Hospital for Sick Children in Toronto, Ontario, said in the statement.

“There have been no approved treatments to date for cholestatic pruritus in Alagille syndrome, and many children ultimately require major surgical interventions such as liver transplantation for refractory pruritus,” she said. “The approval of LIVMARLI signifies a meaningful shift in the treatment paradigm for Alagille syndrome and provides hope for the many families who have lived with persistent itch for far too long.”

The FDA approval is based on the ICONIC study, combined with 5 years of supportive studies that demonstrated significant reductions in pruritus that were maintained for 4 years.

Maralixibat is a minimally absorbed ileal bile acid transporter inhibitor and is the first and only FDA-approved medication for ALGS, a rare liver disease that affects children. ALGS causes abnormalities in bile ducts that can progress into liver disease.

Maralixibat can cause changes in liver tests, fat-soluble vitamin deficiency, and gastrointestinal/intestinal and stomach problems.


U.S. FDA approved 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. Business Wire. News release. September 29, 2021. Accessed September 29, 2021. https://www.businesswire.com/news/home/20210929005810/en/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

Related Videos
Laboratory test tubes and solution with stethoscope background | Image Credit: Shutter2U - stock.adobe.com
Image credit: Andrea Izzotti
Image credit: Krakenimages.com | stock.adobe.com
Human brain digital illustration. Electrical activity, flashes, and lightning on a blue background. | Image Credit: Siarhei - stock.adobe.com
Inflation Reduction Act is shown using the text and the US flag - Image credit: Andrii | stock.adobe.com
3D illustration of heart
Model of a heart
Man experiencing heart pain
© 2023 MJH Life Sciences

All rights reserved.