The US Food and Drug Administration (FDA) has approved maralixibat (Livmarli™) as the first treatment for cholestatic pruritus in patients with Alagille syndrome (ALGS) who are aged 1 year or older, Mirum Pharmaceuticals, Inc. announced. It is now available for prescribing.

Maralixibat, which is administered orally once-daily, is a minimally absorbed ileal bile acid transporter (IBAT) inhibitor. Alagille syndrome, a rare genetic liver disease, affects 2000 to 2500 children in the United States, according to Mirum.

In children with Alagille syndrome, cholestatic pruritus is “serious, unremitting, and debilitating,” said Binita M. Kamath, MBBChir, a pediatric hepatologist at The Hospital for Sick Children (SickKids) in Toronto, Ontario, Canada.

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“Their sleep is disrupted, and they endure bleeding and scarring of the skin due to unrelenting scratching,” she said. “Many children ultimately require major surgical interventions such as liver transplantation for refractory pruritus.”

The approval of maralixibat “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,” Dr. Kamath said.

More on maralixibat’s FDA approval: Relieving the ‘Devastating’ Severe Itching Faced by Patients With Alagille Syndrome

Roberta Smith, president of the Alagille Syndrome Alliance and mother of a child with ALGS, said the approval is “an incredibly meaningful milestone for the ALGS community.”

“Until now, patients have had limited-to-no treatment options to address the severe and unrelenting itch that significantly impacts both patients and their families,” she said.

In the ICONIC clinical trial, maralixibat demonstrated statistically significant reductions in pruritus that were maintained over 4 years, according to Mirum. The approval is based on that study and 5 years of data from supportive studies involving 86 patients with ALGS.

In March, the FDA granted Priority Review to the New Drug Application (NDA) for maralixibat for the treatment of cholestatic pruritus in patients with Alagille syndrome. Mirum recently submitted a marketing authorization application for maralixibat to the European Medicines Agency (EMA) for the treatment of cholestatic disease in Alagille syndrome.

Side effects of maralixibat can include changes in certain liver tests, stomach and intestinal problems, worsening Fat Soluble Vitamin Deficiency, bone fractures, and gastrointestinal bleeding. See the prescribing information for maralixibat here.


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.