Volixibat

Generic Name
Volixibat
Brand Names
-
Drug Type
Small Molecule
Chemical Formula
C38H51N3O12S2
CAS Number
1025216-57-2
Unique Ingredient Identifier
X2JZ0451H8
Background

Volixibat, also known as SHP626 or LUM002, is an investigational drug that will potentially be used for the treatment of Non-Alcoholic Steatohepatitis (NASH). If approved for use, it will be the first available agent for the treatment of NASH.

Volixibat is a selective inhibitor of the apical sodium-dependent bile acid transporter (ASBT), a transmembrane protein primarily expressed by enterocytes of the ileum. Also known as the ileal bile acid transporter (IBAT), ASBT is primarily responsible for the enterohepatic recirculation of bile acids and ultimately for hepatic lipid and glucose metabolism . Inhibiting this enzyme results in a decrease of bile acids returning to the liver, which is helpful for the treatment of NASH as abnormal cholesterol metabolism and accumulation of free cholesterol in the liver have been implicated in its pathogenesis.

According to Shire, the pharmaceutical manufacturer of Volixibat, it has been granted fast track status by the Food and Drug Administration due to promising initial results and a need for therapeutic treatments for NASH.

Indication

Volixibat is an investigational drug that has not been approved for use in any conditions.

Associated Conditions
-
Associated Therapies
-

A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary Cholangitis

Phase 2
Recruiting
Conditions
Interventions
First Posted Date
2021-09-20
Last Posted Date
2024-12-13
Lead Sponsor
Mirum Pharmaceuticals, Inc.
Target Recruit Count
180
Registration Number
NCT05050136
Locations
🇯🇵

Toranomon Hospital, Tokyo, Japan

🇺🇸

Southern California Research Center, Coronado, California, United States

🇺🇸

Science 37, Inc (Remote-homebased Telemedicine), Culver City, California, United States

and more 75 locations

A Placebo-controlled Study of Volixibat in Subjects With Elevated Serum Bile Acids Associated With Intrahepatic Cholestasis of Pregnancy (OHANA)

First Posted Date
2021-01-22
Last Posted Date
2024-08-06
Lead Sponsor
Mirum Pharmaceuticals, Inc.
Target Recruit Count
4
Registration Number
NCT04718961
Locations
🇺🇸

The University of Texas Medical Branch - Galveston, Galveston, Texas, United States

🇺🇸

University of Miami, Miami, Florida, United States

🇺🇸

University of Alabama at Birmingham, Birmingham, Alabama, United States

and more 17 locations

A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC)

Phase 2
Recruiting
Conditions
Interventions
First Posted Date
2020-12-11
Last Posted Date
2024-12-16
Lead Sponsor
Mirum Pharmaceuticals, Inc.
Target Recruit Count
200
Registration Number
NCT04663308
Locations
🇦🇺

University of the Sunshine Coast, Sippy Downs, Australia

🇫🇷

Centre Hospitalier Universitaire Grenoble Alpes (CHU Grenoble Alpes), Grenoble, France

🇨🇦

McGill University Health Centre - Royal Victoria Hospital, Montréal, Canada

and more 87 locations

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

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

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