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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 of MARALIXIBAT CHLORIDE

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

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).

Approval Year

PubMed

PubMed

TitleDatePubMed
A Drug Regimen for Progressive Familial Cholestasis Type 2.
2018 Jan
Placebo-Controlled Randomized Trial of an Intestinal Bile Salt Transport Inhibitor for Pruritus in Alagille Syndrome.
2018 Oct

Sample Use Guides

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 GMT 2023
Edited
by admin
on Fri Dec 15 16:07:01 GMT 2023
Record UNII
V78M04F0XC
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
MARALIXIBAT CHLORIDE
INN   USAN   WHO-DD  
USAN   INN  
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
INN  
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
Created by admin on Fri Dec 15 16:07:01 GMT 2023 , Edited by admin on Fri Dec 15 16:07:01 GMT 2023
EU-Orphan Drug EU/3/13/1217
Created by admin on Fri Dec 15 16:07:01 GMT 2023 , Edited by admin on Fri Dec 15 16:07:01 GMT 2023
FDA ORPHAN DRUG 406113
Created by admin on Fri Dec 15 16:07:01 GMT 2023 , Edited by admin on Fri Dec 15 16:07:01 GMT 2023
FDA ORPHAN DRUG 401513
Created by admin on Fri Dec 15 16:07:01 GMT 2023 , Edited by admin on Fri Dec 15 16:07:01 GMT 2023
Code System Code Type Description
SMS_ID
100000181872
Created by admin on Fri Dec 15 16:07:01 GMT 2023 , Edited by admin on Fri Dec 15 16:07:01 GMT 2023
PRIMARY
RXCUI
2571073
Created by admin on Fri Dec 15 16:07:01 GMT 2023 , Edited by admin on Fri Dec 15 16:07:01 GMT 2023
PRIMARY
EPA CompTox
DTXSID701337104
Created by admin on Fri Dec 15 16:07:01 GMT 2023 , Edited by admin on Fri Dec 15 16:07:01 GMT 2023
PRIMARY
ChEMBL
CHEMBL363392
Created by admin on Fri Dec 15 16:07:01 GMT 2023 , Edited by admin on Fri Dec 15 16:07:01 GMT 2023
PRIMARY
EVMPD
SUB195693
Created by admin on Fri Dec 15 16:07:01 GMT 2023 , Edited by admin on Fri Dec 15 16:07:01 GMT 2023
PRIMARY
FDA UNII
V78M04F0XC
Created by admin on Fri Dec 15 16:07:01 GMT 2023 , Edited by admin on Fri Dec 15 16:07:01 GMT 2023
PRIMARY
USAN
CD-113
Created by admin on Fri Dec 15 16:07:01 GMT 2023 , Edited by admin on Fri Dec 15 16:07:01 GMT 2023
PRIMARY
CAS
228113-66-4
Created by admin on Fri Dec 15 16:07:01 GMT 2023 , Edited by admin on Fri Dec 15 16:07:01 GMT 2023
PRIMARY
PUBCHEM
9831642
Created by admin on Fri Dec 15 16:07:01 GMT 2023 , Edited by admin on Fri Dec 15 16:07:01 GMT 2023
PRIMARY
INN
10073
Created by admin on Fri Dec 15 16:07:01 GMT 2023 , Edited by admin on Fri Dec 15 16:07:01 GMT 2023
PRIMARY
NCI_THESAURUS
C170149
Created by admin on Fri Dec 15 16:07:01 GMT 2023 , Edited by admin on Fri Dec 15 16:07:01 GMT 2023
PRIMARY
DAILYMED
V78M04F0XC
Created by admin on Fri Dec 15 16:07:01 GMT 2023 , Edited by admin on Fri Dec 15 16:07:01 GMT 2023
PRIMARY
Related Record Type Details
PARENT -> SALT/SOLVATE
Related Record Type Details
ACTIVE MOIETY