odevixibat   Click here for help

GtoPdb Ligand ID: 11194

Synonyms: A-4250 | A4250 | Bylvay® | example 5 [US9694018B1] | Unk-D-nTyr-Abu-OH (IUPAC condensed name)
Approved drug
odevixibat is an approved drug (EMA & FDA (2021))
Compound class: Synthetic organic
Comment: Odevixibat is an inhibitor of ileal bile acid transporter (IBAT, or apical sodium-dependent bile acid transporter ASBT; SLC10A2). It was developed by Albireo for potential to treat rare pediatric cholestatic liver diseases, including progressive familial intrahepatic cholestasis (PFIC) and biliary atresia. In July 2021 it became the first drug to be approved as a treament for PFIC-associated pruritus [3].
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Ligand Activity Visualisation Charts

These are box plot that provide a unique visualisation, summarising all the activity data for a ligand taken from ChEMBL and GtoPdb across multiple targets and species. Click on a plot to see the median, interquartile range, low and high data points. A value of zero indicates that no data are available. A separate chart is created for each target, and where possible the algorithm tries to merge ChEMBL and GtoPdb targets by matching them on name and UniProt accession, for each available species. However, please note that inconsistency in naming of targets may lead to data for the same target being reported across multiple charts.

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View interactive charts of activity data across species
2D Structure
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2D Structure

An image of the ligand's 2D structure. For small molecules with SMILES these are drawn using the NCI/CADD Chemical Identifier Resolver. Click on the image to access the chemical structure search tool with the ligand pre-loaded in the structure editor. For other types of ligands, e.g. longer nucleotides and peptides, a manually drawn representation of the molecule may be provided.

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Physico-chemical Properties
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Physico-chemical Properties

Calculated molecular properties are available for small molecules and natural products (not peptides). Properties were generated using the CDK toolkit. All properties were selected to enable the prediction of the Lipinski Rule-of-Five profile or ‘druglikeness’ for each ligand. For more info on each category see the help pages.

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Hydrogen bond acceptors 10
Hydrogen bond donors 5
Rotatable bonds 19
Topological polar surface area 208.05
Molecular weight 740.29
XLogP 6.86
No. Lipinski's rules broken 2
SMILES / InChI / InChIKey
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SMILES / InChI / InChIKey

SMILES (Simplified Molecular Input Line Entry Specification) A specification for unambiguously describing the structure of chemical molecules using short ASCII strings. Canonical SMILES specify a unique representation of the 2D structure without chiral or isotopic specifications. Isomeric SMILES include chiral specification and isotopes.

Standard InChI (IUPAC International Chemical Identifier) and InChIKey InChI is a non-proprietary, standard, textual identifier for chemical substances designed to facilitate linking of information and database searching. An InChIKey is a simplified version of a full InChI, designed for easier web searching.

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Canonical SMILES CCCCC1(CCCC)CN(c2ccccc2)c2c(S(=O)(=O)N1)cc(c(c2)SC)OCC(=O)N[C@@H](C(=O)N[C@H](C(=O)O)CC)c1ccc(cc1)O
Isomeric SMILES CCCCC1(CCCC)CN(c2ccccc2)c2c(S(=O)(=O)N1)cc(c(c2)SC)OCC(=O)N[C@@H](C(=O)N[C@H](C(=O)O)CC)c1ccc(cc1)O
InChI 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
InChI Key XULSCZPZVQIMFM-IPZQJPLYSA-N
No information available.
Summary of Clinical Use Click here for help
Odevixibat was granted EMA orphan drug designation in mid-2012, as a treatment for progressive familial intrahepatic cholestasis (PFIC) [1]. It advanced to Phase 3 clinical evaluations to determine safety and efficacy. In July 2021, both the FDA and the EMA approved odevixibat (Bylvay®) for the treatment of pruritus in all subtypes of PFIC .
Mechanism Of Action and Pharmacodynamic Effects Click here for help
By inhibiting the function of ileal bile acid transporters odevixibat reduces bile-acid re-uptake into liver cells, thus mitigating against the toxic effects of accumulating bile-acids within the liver where disease has disrupted bile transport.
Clinical Trials
Clinical Trial ID Title Type Source Comment References
NCT03566238 This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC 1 or 2 Phase 3 Interventional Albireo
NCT02630875 A4250, an IBAT Inhibitor in Pediatric Cholestasis Phase 2 Interventional Albireo
NCT03659916 Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC Phase 3 Interventional Albireo