The Food and Drug Administration (FDA) has expanded the approval of Livmarli® (maralixibat) to include the treatment of cholestatic pruritus in patients as young as 3 months old with Alagille syndrome. Previously, the treatment had been approved for patients 1 year of age and older.
Livmarli is an ileal bile acid transporter (IBAT) inhibitor. Effectiveness in younger patients was supported by evidence from a study of patients 1 to 15 years of age (N=31) that included 18 weeks of open-label treatment, followed by a 4 week placebo-controlled, randomized withdrawal period, and a subsequent 26-week open-label treatment period.
Safety was established based on data from an open-label, multicenter study (ClinicalTrials.gov Identifier: NCT04729751). Findings showed similar safety, tolerability, and pharmacokinetics in patients 3 to less than 12 months of age and those older than 12 months.
“The vast majority of patients are diagnosed with ALGS before 1 year of age,” said Chris Peetz, president and chief executive officer at Mirum. “The availability of Livmarli will offer an opportunity to introduce a treatment at the beginning of their ALGS journey with the goal of reducing serum bile acids and alleviating the unrelenting burden caused by pruritus.”
The most common adverse reactions reported with Livmarli include 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.
- Mirum announces label expansion for Livmarli in the United States to include infants three months of age and older. News release. Mirum Pharmaceuticals. March 14, 2023. https://www.businesswire.com/news/home/20230313005767/en/Mirum-Announces-Label-Expansion-for-LIVMARLI-in-the-United-States-to-Include-Infants-Three-Months-of-Age-and-Older.
- Livmarli. Package insert. Mirum; 2023. Accessed March 14, 2023. https://files.mirumpharma.com/livmarli/livmarli-prescribinginformation.pdf.