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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 of MARALIXIBAT

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

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
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 Thu Jul 06 14:02:49 UTC 2023
Edited
by admin
on Thu Jul 06 14:02:49 UTC 2023
Record UNII
UYB6UOF69L
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
MARALIXIBAT
USAN   WHO-DD  
USAN  
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
Created by admin on Thu Jul 06 14:02:50 UTC 2023 , Edited by admin on Thu Jul 06 14:02:50 UTC 2023
FDA ORPHAN DRUG 705519
Created by admin on Thu Jul 06 14:02:50 UTC 2023 , Edited by admin on Thu Jul 06 14:02:50 UTC 2023
Code System Code Type Description
EVMPD
SUB195692
Created by admin on Thu Jul 06 14:02:50 UTC 2023 , Edited by admin on Thu Jul 06 14:02:50 UTC 2023
PRIMARY
EPA CompTox
DTXSID001337103
Created by admin on Thu Jul 06 14:02:50 UTC 2023 , Edited by admin on Thu Jul 06 14:02:50 UTC 2023
PRIMARY
NCI_THESAURUS
C170148
Created by admin on Thu Jul 06 14:02:50 UTC 2023 , Edited by admin on Thu Jul 06 14:02:50 UTC 2023
PRIMARY
FDA UNII
UYB6UOF69L
Created by admin on Thu Jul 06 14:02:50 UTC 2023 , Edited by admin on Thu Jul 06 14:02:50 UTC 2023
PRIMARY
DAILYMED
UYB6UOF69L
Created by admin on Thu Jul 06 14:02:50 UTC 2023 , Edited by admin on Thu Jul 06 14:02:50 UTC 2023
PRIMARY
PUBCHEM
9831643
Created by admin on Thu Jul 06 14:02:50 UTC 2023 , Edited by admin on Thu Jul 06 14:02:50 UTC 2023
PRIMARY
SMS_ID
100000181871
Created by admin on Thu Jul 06 14:02:50 UTC 2023 , Edited by admin on Thu Jul 06 14:02:50 UTC 2023
PRIMARY
RXCUI
2571074
Created by admin on Thu Jul 06 14:02:50 UTC 2023 , Edited by admin on Thu Jul 06 14:02:50 UTC 2023
PRIMARY
USAN
CD-112
Created by admin on Thu Jul 06 14:02:50 UTC 2023 , Edited by admin on Thu Jul 06 14:02:50 UTC 2023
PRIMARY
CAS
716313-53-0
Created by admin on Thu Jul 06 14:02:50 UTC 2023 , Edited by admin on Thu Jul 06 14:02:50 UTC 2023
PRIMARY
Related Record Type Details
SALT/SOLVATE -> PARENT
TARGET -> INHIBITOR
Related Record Type Details
ACTIVE MOIETY