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Details

Stereochemistry ABSOLUTE
Molecular Formula C37H48N4O8S2
Molecular Weight 740.9326
Optical Activity UNSPECIFIED
Defined Stereocenters 2 / 2
E/Z Centers 0
Charge 0

SHOW SMILES / InChI
Structure of ODEVIXIBAT

SMILES

CCCCC1(CCCC)CN(c2ccccc2)c3cc(c(cc3S(=O)(=O)N1)OCC(=N[C@]([H])(c4ccc(cc4)O)C(=N[C@@]([H])(CC)C(=O)O)O)O)SC

InChI

InChIKey=XULSCZPZVQIMFM-IPZQJPLYSA-N
InChI=1S/C37H48N4O8S2/c1-5-8-19-37(20-9-6-2)24-41(26-13-11-10-12-14-26)29-21-31(50-4)30(22-32(29)51(47,48)40-37)49-23-33(43)39-34(25-15-17-27(42)18-16-25)35(44)38-28(7-3)36(45)46/h10-18,21-22,28,34,40,42H,5-9,19-20,23-24H2,1-4H3,(H,38,44)(H,39,43)(H,45,46)/t28-,34+/m0/s1

HIDE SMILES / InChI

Molecular Formula C37H48N4O8S2
Molecular Weight 740.9326
Charge 0
Count
Stereochemistry ABSOLUTE
Additional Stereochemistry No
Defined Stereocenters 2 / 2
E/Z Centers 0
Optical Activity UNSPECIFIED

A-4250 (odevixibat) is a selective inhibitor of the ileal bile acid transporter (IBAT) that acts locally in the gut. Ileum absorbs glyco-and taurine-conjugated forms of the bile salts. IBAT is the first step in absorption at the brush-border membrane. A-4250 works by decreasing the re-absorption of bile acids from the small intestine to the liver, whichreduces the toxic levels of bile acids during the progression of the disease. It exhibits therapeutic intervention by checking the transport of bile acids. Studies show that A-4250 has the potential to decrease the damage in the liver cells and the development of fibrosis/cirrhosis of the liver known to occur in progressive familial intrahepatic cholestasis. A-4250 is a designated orphan drug in the USA for October 2012. A-4250 is a designated orphan drug in the EU for October 2016. A-4250 was awarded PRIME status for PFIC by EMA in October 2016. A-4250 is in phase II clinical trials by Albireo for the treatment of primary biliary cirrhosis (PBC) and cholestatic pruritus. In an open label Phase 2 study in children with cholestatic liver disease and pruritus, odevixibat showed reductions in serum bile acids and pruritus in most patients and exhibited a favorable overall tolerability profile.

Approval Year

PubMed

PubMed

TitleDatePubMed
The ileal bile acid transporter inhibitor A4250 decreases serum bile acids by interrupting the enterohepatic circulation.
2016 Jan
Pilot study with IBAT inhibitor A4250 for the treatment of cholestatic pruritus in primary biliary cholangitis.
2018 Apr 27

Sample Use Guides

The primary efficacy objective was to demonstrate the efficacy of once daily dosing of A-4250, 1.5–3 mg orally during a four-week treatment period to relieve patients with primary biliary cholangitis from cholestatic pruritus.
Route of Administration: Oral
Substance Class Chemical
Created
by admin
on Fri Jun 25 23:53:45 UTC 2021
Edited
by admin
on Fri Jun 25 23:53:45 UTC 2021
Record UNII
2W150K0UUC
Record Status Validated (UNII)
Record Version
  • Download
Name Type Language
ODEVIXIBAT
USAN   INN  
Official Name English
BUTANOIC ACID, 2-(((2R)-2-((2-((3,3-DIBUTYL-2,3,4,5-TETRAHYDRO-7-(METHYLTHIO)-1,1-DIOXIDO-5-PHENYL-1,2,5-BENZOTHIADIAZEPIN-8-YL)OXY)ACETYL)AMINO)-2-(4-HYDROXYPHENYL)ACETYL)AMINO)-, (2S)-
Systematic Name English
AZD8294
Code English
A-4250
Code English
AZD-8294
Code English
BUTANOIC ACID, 2-(((2R)-((((3,3-DIBUTYL-2,3,4,5-TETRAHYDRO-7-(METHYLTHIO)-1,1-DIOXIDO-5-PHENYL-1,2,5-BENZOTHIADIAZEPIN-8-YL)OXY)ACETYL)AMINO)(4-HYDROXYPHENYL)ACETYL)AMINO)-, (2S)-
Systematic Name English
ODEVIXIBAT [USAN]
USAN  
Common Name English
A4250
Code English
AR-H064974
Code English
ODEVIXIBAT [INN]
Common Name English
Classification Tree Code System Code
FDA ORPHAN DRUG 658118
Created by admin on Fri Jun 25 23:53:45 UTC 2021 , Edited by admin on Fri Jun 25 23:53:45 UTC 2021
FDA ORPHAN DRUG 370812
Created by admin on Fri Jun 25 23:53:45 UTC 2021 , Edited by admin on Fri Jun 25 23:53:45 UTC 2021
FDA ORPHAN DRUG 371012
Created by admin on Fri Jun 25 23:53:45 UTC 2021 , Edited by admin on Fri Jun 25 23:53:45 UTC 2021
EU-Orphan Drug EU/3/18/2103
Created by admin on Fri Jun 25 23:53:45 UTC 2021 , Edited by admin on Fri Jun 25 23:53:45 UTC 2021
FDA ORPHAN DRUG 370912
Created by admin on Fri Jun 25 23:53:45 UTC 2021 , Edited by admin on Fri Jun 25 23:53:45 UTC 2021
Code System Code Type Description
NCI_THESAURUS
C166598
Created by admin on Fri Jun 25 23:53:45 UTC 2021 , Edited by admin on Fri Jun 25 23:53:45 UTC 2021
PRIMARY
EVMPD
SUB193727
Created by admin on Fri Jun 25 23:53:45 UTC 2021 , Edited by admin on Fri Jun 25 23:53:45 UTC 2021
PRIMARY
PUBCHEM
10153627
Created by admin on Fri Jun 25 23:53:45 UTC 2021 , Edited by admin on Fri Jun 25 23:53:45 UTC 2021
PRIMARY
INN
10706
Created by admin on Fri Jun 25 23:53:45 UTC 2021 , Edited by admin on Fri Jun 25 23:53:45 UTC 2021
PRIMARY
FDA UNII
2W150K0UUC
Created by admin on Fri Jun 25 23:53:45 UTC 2021 , Edited by admin on Fri Jun 25 23:53:45 UTC 2021
PRIMARY
CAS
501692-44-0
Created by admin on Fri Jun 25 23:53:45 UTC 2021 , Edited by admin on Fri Jun 25 23:53:45 UTC 2021
PRIMARY
Related Record Type Details
TARGET -> INHIBITOR
Related Record Type Details
ACTIVE MOIETY
Originator: Albireo; Class: Antipruritic; Mechanism of Action: Sodium-bile acid cotransporter-inhibitor; Orphan Drug Status: Yes for Primary biliary cirrhosis, Intrahepatic cholestasis, Alagille syndrome; On Fast track: No; New Molecular Entity: Yes; Highest Development Phases: Phase II for Intrahepatic cholestasis, Primary biliary cirrhosis, Pruritus - Phase I for Alagille syndrome; Most Recent Events: 25 Jul 2016 Phase-II clinical trials in Intrahepatic cholestasis (PO) (Albireo pipeline, July 2016), 06 Nov 2015 Phase-II clinical trials in Pruritus (In adolescents, In children, In infants) in Germany (PO) (EudraCT2015-001157-32), 21 Aug 2015 Phase-II clinical trials in Pruritus (In children, In infants, In adolescents) in Denmark (PO) (EudraCT2015-001157-32)