MARALIXIBAT

Details

Stereochemistry	ABSOLUTE
Molecular Formula	C40H56N3O4S
Molecular Weight	674.955
Optical Activity	UNSPECIFIED
Defined Stereocenters	2 / 2
E/Z Centers	0
Charge	1

SHOW SMILES / InChI

Structure Search

SMILES

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=STPKWKPURVSAJF-LJEWAXOPSA-N

InChI=1S/C40H56N3O4S/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/q+1/t38-,39-/m1/s1

HIDE SMILES / InChI

Molecular Formula	C40H55N3O4S
Molecular Weight	673.947
Charge	0
Count

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

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

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 `Sat Dec 16 08:35:56 GMT 2023` Edited by `admin` on `Sat Dec 16 08:35:56 GMT 2023`
Record UNII	UYB6UOF69L
Record Status	`Validated (UNII)`
Record Version

Download

Name

Type

Language

MARALIXIBAT

Official Name

English

LUM-001 CATION

Code

English

Maralixibat [WHO-DD]

Common Name

English

LOPIXIBAT

Common Name

English

LUM001 CATION

Code

English

MARALIXIBAT [USAN]

Common Name

English

MARALIXIBAT CATION

Common 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)-

Systematic Name

English

Classification Tree Code System Code

FDA ORPHAN DRUG

406013