FDA Approves Maralixibat (Livmarli) Tablet for Alagille Syndrome, PFIC

The US Food and Drug Administration has approved a new tablet formulation of maralixibat (Livmarli) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).1

According to an April 14, 2025, press release from Mirum Pharmaceuticals, maralixibat tablets are planned to be available in June through Mirum Access Plus. In addition to its already approved indications in ALGS and PFIC, maralixibat is being evaluated in the phase 3 EXPAND study in additional settings of cholestatic pruritus.1

“The approval of LIVMARLI in tablet form provides a meaningful additional treatment option for patients with ALGS and PFIC. It allows flexibility for patients and physicians, with the liquid dosing used by the youngest patients and a convenient one-tablet per dose option for older patients,” said Peter Radovich, president and chief operating officer at Mirum.1 “We have had tremendous success with LIVMARLI since its launch and we hope that the availability of the tablet will provide convenience that positively impacts patients’ lives.”

An orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the FDA in both liquid and tablet formulations for 2 pediatric cholestatic liver diseases. In September 2021, the FDA approved maralixibat for the treatment of cholestatic pruritus in patients with ALGS ≥ 1 year of age, marking the first FDA-approved therapeutic for ALGS. The decision was based on findings from the ICONIC study demonstrating a statistically significant reduction in pruritus maintained through 4 years.1,2

Later, in March 2024, the FDA approved maralixibat for the treatment of cholestatic pruritus in patients ≥ 5 years of age with PFIC. The decision was based on data from the phase 3 MARCH PFIC study, the largest randomized trial conducted in PFIC. Among a cohort of 93 patients with a range of genetic PFIC subtypes including PFIC1, PFIC2, PFIC3, PFIC4, PFIC6, and unidentified mutational status, patients treated with maralixibat had statistically significant improvements in pruritus (P <.0001), serum bile acids (P <.0001), bilirubin (P = .0471), and growth as measured by weight z-score (P = .0391).3

In July 2024, the FDA approved a label expansion to include use of maralixibat in patients with PFIC ≥ 12 months of age as well as the higher concentration formulation evaluated in the phase 3 MARCH study.4

As mentioned in the press release from Mirum, maralixibat is being evaluated in the Phase 3 EXPAND study in additional settings of cholestatic pruritus.1

References

1. Mirum Pharmaceuticals. Mirum’s LIVMARLI Now FDA Approved in Tablet Formulation. April 14, 2025. Accessed April 14, 2025. https://ir.mirumpharma.com/news-events/News/news-details/2025/Mirums-LIVMARLI-Now-FDA-Approved-in-Tablet-Formulation/default.aspx

2. Walter K. FDA Approves First Treatment for Rare Pediatric Liver Disease. September 29, 2021. Accessed April 14, 2025. https://www.hcplive.com/view/fda-approves-first-treatment-rare-pediatric-liver-disease

3. Brooks A. FDA Approval of Maralixibat for Cholestatic Pruritus in PFIC Signals New Frontier for IBAT Inhibitor. March 13, 2024. Accessed April 14, 2025. https://www.hcplive.com/view/fda-approval-of-maralixibat-for-pfic-ibat-inhibitors

4. Brooks A. FDA Approves Label Expansion for Maralixibat (Livmarli) in PFIC. July 25, 2024. Accessed April 14, 2025. https://www.hcplive.com/view/fda-approves-label-expansion-maralixibat-livmarli-pfic