Odevixibat

Summary

Odevixibat (CHEMBL4297588) is an approved small molecule (ATC A05AX05) targeting SLC10A2; indicated across 6 conditions including intrahepatic cholestasis and alagille syndrome.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: A05AX05
• Targets: 1 (SLC10A2)
• Indications: 6 conditions
• Clinical trials: 11
• Chemistry: 740.9 Da · C37H48N4O8S2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4297588
Name	Odevixibat
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	10153627
ATC	A05AX05
Molecular formula	C37H48N4O8S2
Molecular weight	740.9
InChIKey	XULSCZPZVQIMFM-IPZQJPLYSA-N

SMILES: CCCCC1(CN(C2=CC(=C(C=C2S(=O)(=O)N1)OCC(=O)N[C@H](C3=CC=C(C=C3)O)C(=O)N[C@@H](CC)C(=O)O)SC)C4=CC=CC=C4)CCCC

IUPAC name: (2S)-2-[[(2R)-2-[[2-[(3,3-dibutyl-7-methylsulfanyl-1,1-dioxo-5-phenyl-2,4-dihydro-1lambda6,2,5-benzothiadiazepin-8-yl)oxy]acetyl]amino]-2-(4-hydroxyphenyl)acetyl]amino]butanoic acid

Also known as: A-4250, A4250, AR-H064974, AZD-8294, AZD8294, Odevixibat, ODEVIXIBAT

Parent form; salt/anhydrous children: CHEMBL5315120

Patent coverage: 183 distinct patent families (648 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 647 (100%) of the total. Mentions count patents naming the compound (not distinct inventions), so promiscuous / reference molecules inflate the mention figure — families are the dedup metric.

Targets

Targets

Primary targets (GtoPdb curated mechanism): the Cancer dependency column is the DepMap CRISPR fitness signal (% of screened cell lines dependent on the target).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
SLC10A2	Sodium/bile acid and sulphated solute cotransporter 2	Inhibition	9.8	0%	Q12908

Broader ChEMBL bioactivity targets: 1 (assay-derived). Sample: Bile acid receptor.

Bioactivity

ChEMBL activities: 1 potent at pChembl ≥ 5 of 1 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
NR1H4	9.8	IC50	0.16	nM	CHEMBL_ACT_26686508

Target pathways

Aggregated over 1 target gene(s): SLC10A2.

Top Reactome pathways

1 total, by targets touching each:

Pathway	Targets	Genes
Recycling of bile acids and salts	1	SLC10A2

Dominant GO biological processes

GO term	Targets
response to bacterium	1
bile acid and bile salt transport	1
monoatomic ion transport	1
sodium ion transport	1
lipid transport	1
transmembrane transport	1

Indications & clinical

Indications

6 indications (1 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
intrahepatic cholestasis	4	MONDO:0019072	MONDO:0019072
Alagille syndrome	3	MONDO:0007318	MONDO:0007318
biliary atresia	3	MONDO:0008867	MONDO:0008867
primary biliary cholangitis	2	MONDO:0005388	EFO:0004267
metabolic dysfunction-associated steatohepatitis	2	MONDO:0007027	EFO:1001249
cholestasis	2	MONDO:0001751	MONDO:0001751

Clinical trials

Total trials: 11.

Phase distribution

Phase	Trials
PHASE3	6
PHASE2	2
PHASE1	2
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04336722	PHASE3	ACTIVE_NOT_RECRUITING	Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)
NCT05035030	PHASE3	RECRUITING	Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome
NCT05426733	PHASE3	ENROLLING_BY_INVITATION	An Open-label Extension Study to Evaluate Long-term Efficacy and Safety of Odevixibat in Children With Biliary Atresia
NCT03566238	PHASE3	COMPLETED	This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC Types 1 or 2
NCT03659916	PHASE3	COMPLETED	Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC
NCT04674761	PHASE3	COMPLETED	Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome
NCT02360852	PHASE2	TERMINATED	IBAT Inhibitor A4250 for Cholestatic Pruritus
NCT02630875	PHASE2	COMPLETED	A4250, an IBAT Inhibitor in Pediatric Cholestasis
NCT02963077	PHASE1	COMPLETED	A Safety and Pharmakokinetic Study of A4250 Alone or in Combination With A3384
NCT03608319	PHASE1	COMPLETED	Study of A4250 in Healthy Volunteers Under Fasting, Fed and Sprinkled Conditions
NCT04483531	Not specified	APPROVED_FOR_MARKETING	Odevixibat for the Treatment of Progressive Familial Intrahepatic Cholestasis

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline or drug-level clinical/variant annotations in PharmGKB for this molecule.

Related molecules

Related molecules

Molecules sharing ≥1 of this drug’s curated primary targets, merged from two biobtree sources and ranked by shared-target count, then clinical phase: ChEMBL clinical-stage candidates (development phase ≥2) and PubChem drug-class bioactivity (approved / known drugs acting on the target). Deduplicated by drug name; the drug’s own salt forms are excluded. Note: for a drug with few primary targets a shared-target match can reflect off-target / promiscuous binding rather than the same therapeutic mechanism — the phase ordering surfaces bona-fide therapeutics first.

9 molecules share ≥1 primary target. Top 9 by shared-target count:

Molecule	Source	Status	Shared targets
CHENODIOL	ChEMBL + PubChem	Phase 4 (approved)	SLC10A2
CYCLOSPORINE	ChEMBL + PubChem	Phase 4 (approved)	SLC10A2
DEOXYCHOLIC ACID	ChEMBL + PubChem	Phase 4 (approved)	SLC10A2
MARALIXIBAT	ChEMBL + PubChem	Phase 4 (approved)	SLC10A2
MARALIXIBAT CHLORIDE	ChEMBL + PubChem	Phase 4 (approved)	SLC10A2
URSODIOL	ChEMBL + PubChem	Phase 4 (approved)	SLC10A2
TAURURSODIOL	ChEMBL	Phase 4 (approved)	SLC10A2
LINERIXIBAT	ChEMBL	Phase 3	SLC10A2
TAURODEOXYCHOLIC ACID	ChEMBL	Phase 2	SLC10A2