Maralixibat
- Generic Name
- Maralixibat
- Brand Names
- Livmarli
- Drug Type
- Small Molecule
- Chemical Formula
- C40H56N3O4S
- 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
- Associated Therapies
- -
Evaluation of Maralixibat in Pruritus Associated with General Cholestatic Liver Disease (EXPAND)
- Conditions
- Interventions
- First Posted Date
- 2024-08-14
- Last Posted Date
- 2024-12-11
- Lead Sponsor
- Mirum Pharmaceuticals, Inc.
- Target Recruit Count
- 90
- Registration Number
- NCT06553768
- Locations
- 🇫🇷
Hôpitaux Universitaires de Marseille Timone, Marseille, France
🇮🇹ISMETT - Istituto Mediterraneo per i Trapianti e Terapie ad Alta Specializzazione, Palermo, Italy
🇺🇸Children's Hospital Los Angeles (CHLA), Los Angeles, California, United States
Long-Term Safety and Clinical Outcomes of Livmarli in Patients With Alagille Syndrome (LEAP)
- First Posted Date
- 2024-01-05
- Last Posted Date
- 2024-03-12
- Lead Sponsor
- Mirum Pharmaceuticals, Inc.
- Target Recruit Count
- 50
- Registration Number
- NCT06193928
- Locations
- 🇺🇸
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 Mercy Kansas City, Department of Gastroenterology, Section of Hepatology, Kansas City, Missouri, United States
🇺🇸Children Hospital of Pittsburgh, Pittsburgh, Pennsylvania, United States
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).
- Conditions
- Interventions
- First Posted Date
- 2021-01-28
- Last Posted Date
- 2024-03-15
- Lead Sponsor
- Mirum Pharmaceuticals, Inc.
- Target Recruit Count
- 12
- 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
A Maralixibat Expanded Access Program for Patients With Cholestatic Pruritus Associated With Alagille Syndrome (ALGS)
- Conditions
- 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
Evaluation of Maralixibat in Biliary Atresia Response Post-Kasai
- First Posted Date
- 2020-08-24
- Last Posted Date
- 2024-03-08
- 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
An Extension Study of Maralixibat in Patients with Progressive Familial Intrahepatic Cholestasis (PFIC)
- Conditions
- Interventions
- First Posted Date
- 2019-12-04
- Last Posted Date
- 2024-10-03
- 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
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
- First Posted Date
- 2019-11-19
- Last Posted Date
- 2024-01-30
- 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
A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial Intrahepatic Cholestasis (MARCH-PFIC)
- Conditions
- Interventions
- 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
A Placebo-controlled Study of Maralixibat (SHP625) in Pediatric Subjects With Progressive Familial Intrahepatic Cholestasis (PFIC)
- Conditions
- Interventions
- First Posted Date
- 2017-11-27
- Last Posted Date
- 2019-03-18
- Lead Sponsor
- Mirum Pharmaceuticals, Inc.
- Registration Number
- NCT03353454
Study to Assess the Relative Potency of Multiple Oral Doses of LUM001 and SHP626 in Overweight and Obese Adults as Assessed by Fecal Bile Acid Excretion
- Conditions
- Interventions
- First Posted Date
- 2015-06-18
- Last Posted Date
- 2019-04-04
- Lead Sponsor
- Mirum Pharmaceuticals, Inc.
- Target Recruit Count
- 84
- Registration Number
- NCT02475317
- Locations
- 🇺🇸
New Orleans Center for Clinical Research, Knoxville, Tennessee, United States
Drug Development Updates
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