Maralixibat

Generic Name: Maralixibat

Brand Names: Livmarli

Drug Type: Small Molecule

Chemical Formula: C_₄₀H_₅₆N_₃O_₄S

CAS Number: 716313-53-0

Unique Ingredient Identifier: UYB6UOF69L

Background: Maralixibat (also known as SHP625, LUM001, and lopixibat) is an ileal bile acid transporter inhibitor, like odevixibat. Maralixibat is indicated for the treatment of cholestatic pruritus in patients with Alagille syndrome.

Previously, patients with cholestatic pruritus associated with Alagille syndrome were treated with antihistamines, rifampin, ursodeoxycholic acid, cholestyramine, naltrexone, and sertraline alone or in combination. No clinical trials have been performed to assess the efficacy of these treatments for cholestatic pruritus and treatments were given based on a prescriber's clinical experience. Surgical interventions such as partial external bile diversion and ileal exclusion have also been used as treatments.

Maralixibat represents the first FDA-approved treatment for cholestatic pruritus in patients with Alagille syndrome. It was granted FDA approval on 29 September 2021. In October 2022, the EMA's Committee for Medicinal Products for Human Use (CHMP) recommended maralixibat be granted marketing authorization for the treatment of cholestatic pruritus in patients with Alagille syndrome: it was granted marketing authorization in Europe on 13 December 2022. On July 21, 2023, maralixibat was also approved by Health Canada.

Indication: Maralixibat is indicated in the treatment of cholestatic pruritus in patients with Alagille syndrome. It is approved for use in patients at least one-month-old in the US and at least two months old in Europe. In Canada, it is reserved for use in adults.

Associated Conditions: Cholestatic pruritus

Clinical Trials

Evaluation of Maralixibat in Pruritus Associated With General Cholestatic Liver Disease (EXPAND)

Phase 3

Recruiting

Conditions: Cholestatic Liver Disease (Except ALGS, PFIC, PBC and PSC)

Interventions: Other: Placebo
Drug: Maralixibat

First Posted Date: 2024-08-14

Last Posted Date: 2025-05-01

Lead Sponsor: Mirum Pharmaceuticals, Inc.

Target Recruit Count: 90

Registration Number: NCT06553768

Locations: 🇺🇸
Children's Hospital Los Angeles (CHLA), Los Angeles, California, United States
🇺🇸
Stanford Children's Health in Palo Alto, Palo Alto, California, United States
🇺🇸
Lurie Children's Hospital, Chicago, Illinois, United States

and more 29 locations

Maralixibat in Patients With Cystic Fibrosis and Constipation, A Within-Subjects Pilot Study

Phase 2

Not yet recruiting

Conditions: Constipation Chronic Idiopathic
Cystic Fibrosis

Interventions: Drug: Maralixibat 9.5 MG/ML [Livmarli]

First Posted Date: 2024-05-14

Last Posted Date: 2024-05-14

Lead Sponsor: Children's Hospital Los Angeles

Target Recruit Count: 20

Registration Number: NCT06413368

Locations: 🇺🇸
Children's Hospital Los Angeles, Los Angeles, California, United States

Long-Term Safety and Clinical Outcomes of Livmarli in Patients With Alagille Syndrome (LEAP)

Recruiting

Conditions: Alagille Syndrome

Interventions: Drug: Maralixibat

First Posted Date: 2024-01-05

Last Posted Date: 2024-12-27

Lead Sponsor: Mirum Pharmaceuticals, Inc.

Target Recruit Count: 50

Registration Number: NCT06193928

Locations: 🇺🇸
Children's Hospital Los Angeles CHLA, Los Angeles, California, United States
🇺🇸
Section of Gastroenterology, Hepatology and Nutrition, Department of Pediatrics and the Digestive Health Institute, Children's Hospital of Colorado and University of Colorado, Aurora, Colorado, United States
🇺🇸
Children's Healthcare of Atlanta - Emory University School of Medicine, Atlanta, Georgia, United States

and more 5 locations

A Study to Evaluate the Safety and Tolerability of Maralixibat in Infant Participants With Cholestatic Liver Diseases Including Progressive Familial Intrahepatic Cholestasis (PFIC) and Alagille Syndrome (ALGS).

Phase 2

Completed

Conditions: Alagille Syndrome
Progressive Familial Intrahepatic Cholestasis
Cholestatic Liver Disease

Interventions: Drug: Maralixibat

First Posted Date: 2021-01-28

Last Posted Date: 2025-02-06

Lead Sponsor: Mirum Pharmaceuticals, Inc.

Target Recruit Count: 27

Registration Number: NCT04729751

Locations: 🇵🇱
Instytut Pomnik-Centrum Zdrowia Dziecka, Warsaw, Poland
🇬🇧
King's College Hospital, London, United Kingdom
🇺🇸
University of California - San Francisco, San Francisco, California, United States

and more 11 locations

A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)

Conditions: Alagille Syndrome

First Posted Date: 2020-08-28

Last Posted Date: 2021-10-20

Lead Sponsor: Mirum Pharmaceuticals, Inc.

Registration Number: NCT04530994

Locations: 🇺🇸
Georgetown University, Washington, District of Columbia, United States
🇺🇸
Ochsner Clinic Foundation, New Orleans, Louisiana, United States
🇺🇸
Medical College of Wisconsin, Milwaukee, Wisconsin, United States

and more 11 locations

Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai

Phase 2

Completed

Conditions: Biliary Atresia

Interventions: Drug: Maralixibat
Other: Placebo

First Posted Date: 2020-08-24

Last Posted Date: 2025-03-19

Lead Sponsor: Mirum Pharmaceuticals, Inc.

Target Recruit Count: 75

Registration Number: NCT04524390

Locations: 🇻🇳
Vietnam National Children's Hospital, Hanoi, Vietnam
🇨🇳
Children's Hospital of Fudan University, Shanghai, China
🇨🇳
The Children's Hospital, Zhejiang University School of Medicine, Hanzhou, Zhejiang, China

and more 19 locations

An Extension Study of Maralixibat in Patients with Progressive Familial Intrahepatic Cholestasis (PFIC)

Phase 3

Active, not recruiting

Conditions: Progressive Familial Intrahepatic Cholestasis (PFIC)

Interventions: Drug: Maralixibat

First Posted Date: 2019-12-04

Last Posted Date: 2025-02-14

Lead Sponsor: Mirum Pharmaceuticals, Inc.

Target Recruit Count: 90

Registration Number: NCT04185363

Locations: 🇸🇬
KK Women's and Children's Hospital, Singapore, Singapore
🇺🇸
Children's Hospital Los Angeles, Los Angeles, California, United States
🇺🇸
Medstar Georgetown University Hospital, Washington, District of Columbia, United States

and more 25 locations

MRX-800: A Long-Term Safety Study of Maralixibat in the Treatment of Cholestatic Liver Disease in Subjects Who Previously Participated in a Maralixibat Study

Phase 2

Completed

Conditions: Cholestatic Liver Disease

Interventions: Drug: Maralixibat

First Posted Date: 2019-11-19

Last Posted Date: 2025-03-06

Lead Sponsor: Mirum Pharmaceuticals, Inc.

Target Recruit Count: 52

Registration Number: NCT04168385

Locations: 🇺🇸
Children's Hospital of Philadelphia, Philadelphia, Pennsylvania, United States
🇵🇱
The Children's Memorial Health Institute, Warsaw, Poland
🇺🇸
Children'S Hospital Los Angeles, Los Angeles, California, United States

and more 14 locations

A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)

Phase 3

Completed

Conditions: Progressive Familial Intrahepatic Cholestasis (PFIC)

Interventions: Drug: Maralixibat
Other: Placebo

First Posted Date: 2019-04-05

Last Posted Date: 2023-12-11

Lead Sponsor: Mirum Pharmaceuticals, Inc.

Target Recruit Count: 93

Registration Number: NCT03905330

Locations: 🇩🇪
Medizinische Hochschule Hannover, Hanover, Germany
🇺🇸
Cincinnati Children's Hospital, Cincinnati, Ohio, United States
🇫🇷
Hospices Civils de Lyon - Hopital Femme Mere Enfant Service de Gastroenterologie, Hepatologie et Nutrition, Lyon, France

and more 28 locations

A Placebo-controlled Study of Maralixibat (SHP625) in Pediatric Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)

Phase 3

Withdrawn

Conditions: Progressive Familial Intrahepatic Cholestasis (PFIC)

Interventions: Drug: Maralixibat
Drug: Placebo

First Posted Date: 2017-11-27

Last Posted Date: 2019-03-18

Lead Sponsor: Mirum Pharmaceuticals, Inc.

Registration Number: NCT03353454

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