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
-
Clinical Trials
Related News
finance.yahoo.com
·

GSK Drug for Rare Disease-Related Itch Meets Goal in Phase III Study

GSK's phase III GLISTEN study shows linerixibat significantly improves cholestatic pruritus in PBC patients, with potential to be the first global therapy for PBC-related itch. Preliminary safety data aligns with previous studies. Full data to be presented at a future medical conference.
hcplive.com
·

Hepatology Month in Review: October 2024

October 2024 saw active hepatic pipeline with FDA Breakthrough Therapy Designations for volixibat and survodutide, and an extended review for obeticholic acid. Research on hepatitis treatment strategies to meet WHO elimination goals was prominent at the ACG 2024 meeting. Key topics included HBV treatment eligibility, universal HCV screening, and point-of-care HCV RNA testing. ACG 2024 also featured discussions on alcohol use disorder pharmacotherapy, seladelpar efficacy in PBC, and elafibranor's safety and efficacy in advanced PBC.
liverdiseasenews.com
·

FDA names volixibat, treating pruritis in PBC, breakthrough therapy

Mirum Pharmaceuticals' volixibat granted breakthrough therapy status by FDA for pruritus in PBC, based on Phase 2b VANTAGE trial interim results showing significant itch reduction. Trial now in confirmatory phase, aiming to support regulatory approval. Volixibat works by inhibiting ileal bile acid transporter to reduce bile acid accumulation and alleviate PBC symptoms.

Related Clinical Trials:

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

Mirum Pharmaceuticals' Volixibat Garners Buy Rating Amidst Breakthrough Therapy

Ed Arce of H.C. Wainwright maintains Buy rating on Mirum Pharmaceuticals (MIRM) with $66.00 price target, citing FDA's Breakthrough Therapy Designation for volixibat in treating cholestatic pruritus in PBC patients, and its potential to offer substantial improvement over existing therapies.
pharmaceutical-business-review.com
·

Volixibat gains FDA breakthrough therapy designation

FDA grants breakthrough therapy designation to Mirum's volixibat for treating cholestatic pruritus in PBC patients. Volixibat, under Phase IIb VANTAGE study, showed significant pruritus reduction and 75% patient improvement in serum bile acids. The drug selectively inhibits ileal bile acid transporter, reducing systemic bile acids. The confirmatory VANTAGE study is ongoing, with full enrolment expected by 2026.
placera.se
·

Volixibat Granted Breakthrough Therapy Designation for Cholestatic Pruritus in Primary

FDA grants Breakthrough Therapy Designation to Mirum's volixibat for cholestatic pruritus in PBC, based on Phase 2b VANTAGE study's positive interim analysis. Confirmatory study ongoing, with enrollment completion expected in 2026.
empr.com
·

Volixibat Granted Breakthrough Therapy Status for PBC-Related Itching

FDA grants Breakthrough Therapy designation to volixibat for treating cholestatic pruritus in primary biliary cholangitis patients. Volixibat, an ileal bile acid transporter inhibitor, showed significant itch reduction in phase 2b VANTAGE study. Mild to moderate diarrhea was the most common adverse event. VANTAGE trial continues with 20mg twice daily dosing, aiming for completion in 2026.

Related Clinical Trials:

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

Volixibat Granted Breakthrough Therapy Designation for Cholestatic Pruritus in Primary Biliary Cholangitis

The FDA granted Breakthrough Therapy Designation to volixibat for treating cholestatic pruritus in primary biliary cholangitis (PBC) patients, based on positive Phase 2b VANTAGE study results. The study's confirmatory phase is ongoing, with enrollment expected to complete in 2026.
pharmacytimes.com
·

FDA Grants Breakthrough Therapy Deignation for Volixibat in Cholestatic Pruritus

The FDA granted breakthrough therapy designation to volixibat, an oral ileal bile acid transporter inhibitor, for treating cholestatic pruritus in primary biliary cholangitis (PBC) patients. The designation is based on the phase 2b VANTAGE study's interim analysis, showing statistically significant improvements in pruritus and serum bile acids, with positive safety signals. Confirmatory trial completion is expected in 2026.

Related Clinical Trials:

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

FDA grants breakthrough status to Mirum's PBC drug

FDA awards Breakthrough Therapy Designation to Mirum Pharmaceuticals' volixibat for primary biliary cholangitis (PBC), based on Phase 2b VANTAGE study interim results showing significant improvement in cholestatic pruritus. Volixibat, an oral medication inhibiting ileal bile acid transporter (IBAT), aims to reduce systemic bile acids, potentially offering a novel treatment for adult cholestatic diseases. Full VANTAGE study results expected by 2026.
© Copyright 2024. All Rights Reserved by MedPath
HomeTerms & ConditionsPrivacy Policy