MARALIXIBAT CHLORIDE

Details

Stereochemistry	ABSOLUTE
Molecular Formula	C40H56N3O4S.Cl
Molecular Weight	710.408
Optical Activity	UNSPECIFIED
Defined Stereocenters	2 / 2
E/Z Centers	0
Charge	0

SHOW SMILES / InChI

Structure Search

SMILES

[Cl-].CCCCC1(CCCC)CS(=O)(=O)C2=CC=C(C=C2[C@H]([C@H]1O)C3=CC=C(OCC4=CC=C(C[N+]56CCN(CC5)CC6)C=C4)C=C3)N(C)C

InChI

InChIKey=POMVPJBWDDJCMP-RUKDTIIFSA-M

InChI=1S/C40H56N3O4S.ClH/c1-5-7-19-40(20-8-6-2)30-48(45,46)37-18-15-34(41(3)4)27-36(37)38(39(40)44)33-13-16-35(17-14-33)47-29-32-11-9-31(10-12-32)28-43-24-21-42(22-25-43)23-26-43;/h9-18,27,38-39,44H,5-8,19-26,28-30H2,1-4H3;1H/q+1;/p-1/t38-,39-;/m1./s1

HIDE SMILES / InChI

Molecular Formula	C40H56N3O4S
Molecular Weight	674.955
Charge	1
Count

Stereochemistry	ABSOLUTE
Additional Stereochemistry	No
Defined Stereocenters	2 / 2
E/Z Centers	0
Optical Activity	UNSPECIFIED

Molecular Formula	Cl
Molecular Weight	35.453
Charge	-1
Count

Stereochemistry	ACHIRAL
Additional Stereochemistry	No
Defined Stereocenters	0 / 0
E/Z Centers	0
Optical Activity	NONE

Description
Sources: https://mirumpharma.com/#toggle-id-5 | https://www.ncbi.nlm.nih.gov/pubmed/30859149 | https://www.ncbi.nlm.nih.gov/pubmed/30288474

Maralixibat (SHP625; formerly LUM001 or lopixibat) is a potent, apical, sodium‐dependent, bile acid transporter competitive inhibitor with minimal systemic absorption. Maralixibat works by reducing systemic levels of bile acids. In animal models of cholestasis, maralixibat blocked reabsorption of bile acids in the terminal ileum, thereby reducing enterohepatic recirculation to the liver and increasing fecal excretion of bile acids. Maralixibat is being evaluated as a treatment for children with rare cholestatic liver diseases, including Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).

Originator

Approval Year

PubMed

Title	Date	PubMed
Placebo-Controlled Randomized Trial of an Intestinal Bile Salt Transport Inhibitor for Pruritus in Alagille Syndrome.	2018 Oct	30288474
A Randomized, Controlled, Phase 2 Study of Maralixibat in the Treatment of Itching Associated With Primary Biliary Cholangitis.	2019 Mar	30859149

Sample Use Guides

In Vivo Use Guide

Phase 3 study to evaluate the efficacy and safety of maralixibat in subjects with progressive familial intrahepatic cholestasis. Participants will be randomized to Maralixibat oral solution (up to 600 microgram per kilogram [mcg/kg]) orally twice daily for 26 weeks.

Route of Administration: Oral

Substance Class	Chemical Created by `admin` on `Fri Dec 15 16:07:01 UTC 2023` Edited by `admin` on `Fri Dec 15 16:07:01 UTC 2023`
Record UNII	V78M04F0XC
Record Status	`Validated (UNII)`
Record Version

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Name

Type

Language

MARALIXIBAT CHLORIDE

Official Name

English

4-AZA-1-AZONIABICYCLO(2.2.2)OCTANE, 1-((4-((4-((4R,5R)-3,3-DIBUTYL-7-(DIMETHYLAMINO)-2,3,4,5-TETRAHYDRO-4-HYDROXY-1,1-DIOXIDO-1-BENZOTHIEPIN-5-YL)PHENOXY)METHYL)PHENYL)METHYL)-, CHLORIDE (1:1)

Systematic Name

English

LOPIXIBAT CHLORIDE

Official Name

English

MARALIXIBAT CHLORIDE [USAN]

Common Name

English

LIVMARLI

Brand Name

English

LUM-001

Code

English

MARALIXIBAT CHLORIDE [ORANGE BOOK]

Common Name

English

LUM001

Code

English

maralixibat chloride [INN]

Common Name

English

Maralixibat chloride [WHO-DD]

Common Name

English

Classification Tree Code System Code

FDA ORPHAN DRUG

406213