Deoxycholic Acid

Summary

Deoxycholic Acid (CHEMBL406393) is an approved small molecule (ATC D11AX24) targeting FPR1, GPBAR1, and NR1H4; indicated across 5 conditions including lipoma and neurofibroma.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: D11AX24
• Targets: 3 (FPR1, GPBAR1, NR1H4)
• Indications: 5 conditions
• Clinical trials: 29
• Chemistry: 392.6 Da · C24H40O4

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL406393
Name	Deoxycholic Acid
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	222528
ChEBI	CHEBI:28834
ATC	D11AX24
Molecular formula	C24H40O4
Molecular weight	392.6
InChIKey	KXGVEGMKQFWNSR-LLQZFEROSA-N

SMILES: C[C@H](CCC(=O)O)[C@H]1CC[C@@H]2[C@@]1([C@H](C[C@H]3[C@H]2CC[C@H]4[C@@]3(CC[C@H](C4)O)C)O)C

IUPAC name: (4R)-4-[(3R,5R,8R,9S,10S,12S,13R,14S,17R)-3,12-dihydroxy-10,13-dimethyl-2,3,4,5,6,7,8,9,11,12,14,15,16,17-tetradecahydro-1H-cyclopenta[a]phenanthren-17-yl]pentanoic acid

ChEBI definition: A bile acid that is 5β-cholan-24-oic acid substituted by hydroxy groups at positions 3 and 12 respectively.

Other ChEBI roles (chemical / environmental): human blood serum metabolite.

Also known as: Acide desoxycholique, Acido desoxicolico, ATX-101, ATX-101 (DEOXYCHOLIC ACID), Deoxycholic acid, Desoxycholic acid, DWJ-211, DWJ211, Kybella, NSC-8797, deoxycholic acid, Deoxycholate

Parent form; salt/anhydrous children: CHEMBL1365278, CHEMBL1884070

Patent coverage: 19,746 distinct patent families (63,215 SureChEMBL compound mentions), from 1 matched compound structure(s). 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
FPR1	FPR1	Antagonist	4	0%	P21462
GPBAR1	GPBA receptor	Full agonist	6.2	0.6%	Q8TDU6
NR1H4	Farnesoid X receptor	Agonist	4	0.7%	Q96RI1

Broader ChEMBL bioactivity targets: 6 (assay-derived). Sample: Bile acid receptor, Thromboxane A2 receptor, Steroid Delta-isomerase, cGMP-inhibited 3’,5’-cyclic phosphodiesterase 3A, Ileal sodium/bile acid cotransporter, G-protein coupled bile acid receptor 1.

Bioactivity

ChEMBL activities: 5 potent at pChembl ≥ 5 of 11 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
GPBAR1	6	EC50	1010	nM	CHEMBL_ACT_25994697
GPBAR1	6	EC50	1010	nM	CHEMBL_ACT_29078710
GPBAR1	5.9	EC50	1250	nM	CHEMBL_ACT_2117892
SLC10A2	5.2	Ki	6300	nM	CHEMBL_ACT_11003216
SLC10A2	5.2	Ki	6300	nM	CHEMBL_ACT_11003217

Target pathways

Aggregated over 3 target gene(s): FPR1, GPBAR1, NR1H4.

Top Reactome pathways

14 total, by targets touching each:

Pathway	Targets	Genes
Recycling of bile acids and salts	1	NR1H4
Synthesis of bile acids and bile salts	1	NR1H4
Synthesis of bile acids and bile salts via 7alpha-hydroxycholesterol	1	NR1H4
Synthesis of bile acids and bile salts via 27-hydroxycholesterol	1	NR1H4
PPARA activates gene expression	1	NR1H4
Endogenous sterols	1	NR1H4
Class A/1 (Rhodopsin-like receptors)	1	GPBAR1
Nuclear Receptor transcription pathway	1	NR1H4
SUMOylation of intracellular receptors	1	NR1H4
G alpha (s) signalling events	1	GPBAR1
G alpha (i) signalling events	1	FPR1
Formyl peptide receptors bind formyl peptides and many other ligands	1	FPR1
Interleukin-10 signaling	1	FPR1
Neutrophil degranulation	1	FPR1

Dominant GO biological processes

GO term	Targets
inflammatory response	2
signal transduction	2
G protein-coupled receptor signaling pathway	2
cellular response to bile acid	2
complement receptor mediated signaling pathway	1
chemotaxis	1
adenylate cyclase-modulating G protein-coupled receptor signaling pathway	1
phospholipase C-activating G protein-coupled receptor signaling pathway	1
positive regulation of cytosolic calcium ion concentration	1
obsolete nitric oxide mediated signal transduction	1
cell communication	1
signaling	1
adenylate cyclase-activating G protein-coupled bile acid receptor signaling pathway	1
positive regulation of ERK1 and ERK2 cascade	1
energy homeostasis	1

Indications & clinical

Indications

5 indications (0 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
lipoma	2	MONDO:0005106	EFO:0000759
neurofibroma	1	MONDO:0016755	EFO:0000622
neurofibromatosis type 1	1	MONDO:0018975	MONDO:0018975
obesity disorder	0	MONDO:0011122	EFO:0001073

1 further indication record had no mapped disease name (EFO/MeSH-only) or were duplicates, and are omitted.

Clinical trials

Total trials: 29.

Phase distribution

Phase	Trials
PHASE1	11
PHASE3	5
PHASE2	4
Not specified	4
PHASE4	3
PHASE1/PHASE2	1
EARLY_PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT04089722	PHASE4	COMPLETED	Efficacy and Safety of Deoxycholic Acid Injections for Removal of Adipose Tissue in the Bra Strap Fat Region
NCT04841551	PHASE4	COMPLETED	Kybella for the Treatment of Flank Fat (FF)
NCT06783621	PHASE4	COMPLETED	A Study to Evaluate Patient Satisfaction With the Overall Face and Neck Appearance After Combined Treatment of OnabotulinumtoxinA, JUVÉDERM® Products, KYBELLA, CoolSculpting Elite and SkinMedica Products
NCT00361517	PHASE3	COMPLETED	To Determine Whether Galactomannan Test Will Help to Detect Fungal Infections Early and Hence Start Treatment Early
NCT02035267	PHASE3	COMPLETED	Safety Study of ATX-101 (Deoxycholic Acid) in Subjects With Mild or Extreme Fullness of Submental Fat
NCT02123134	PHASE3	COMPLETED	Safety Study of Deoxycholic Acid for the Reduction of Localized Subcutaneous Fat in the Submental Area in Subjects 65 to 75 Years of Age
NCT02163902	PHASE3	COMPLETED	Long-term Follow-up Study on Safety and Maintenance of Efficacy of ATX-101
NCT03946592	PHASE3	COMPLETED	Evaluate the Efficacy and Safety of DWJ211 in Patient With Moderate or Severe Submental Fat
NCT02159729	PHASE2	COMPLETED	Long Term Follow-up Study on Safety and Maintenance of Efficacy of ATX-101
NCT03224117	PHASE2	COMPLETED	Clinicial Trial to Evaluate the Efficacy and Safety of DWJ211 in Patient With Moderate or Severe Submental Fat
NCT04814875	PHASE1/PHASE2	TERMINATED	A Study to Evaluate the Combination of ATX-101 and Platinum-based Chemotherapy
NCT05116683	PHASE2	TERMINATED	ATX-101 in Advanced Dedifferentiated Liposarcoma and Leiomyosarcoma
NCT05260008	PHASE2	TERMINATED	Study Assessing Pain Relief After Replacement of the Knee
NCT06300502	PHASE1	ENROLLING_BY_INVITATION	Assessing the Efficacy of Repeat, Monthly Treatments of Deoxycholate for NF1 Associated Cutaneous Neurofibromas (cNFs)
NCT07237425	PHASE1	RECRUITING	Intralesional Ronkyla Plus Injection for the Treatment of Superficial Lipoma
NCT00618709	PHASE1	COMPLETED	Dose-Escalation Safety and Pharmacokinetic Study of ATX-101
NCT00835952	PHASE1	COMPLETED	ATX-101 Abdominoplasty
NCT01319916	PHASE1	COMPLETED	Lipid and Adipokine Profiles Following Subcutaneous Injection of ATX-101 Into Abdominal Fat Tissue
NCT01462786	PHASE1	COMPLETED	To Evaluate the Safety, Tolerability and to Compare the Pharmacokinetic Profile of ATX-101
NCT01706679	PHASE1	COMPLETED	Evaluation of the Effect of ATX-101 on QT/QTc Intervals
NCT04159961	PHASE1	COMPLETED	Pharmacokinetics and Safety, Tolerability of DWJ211in Healthy Volunteers
NCT04730583	PHASE1	COMPLETED	Tolerability of Device Based Therapies for Neurofibromatosis Type 1 Cutaneous Neurofibromas
NCT06120036	PHASE1	COMPLETED	Dosing and Tolerability of Deoxycholic Acid vs. Polidocanol in the Treatment of Neurofibromatosis Type 1 Cutaneous Neurofibromas
NCT06132165	PHASE1	COMPLETED	Efficacy of Skin Cooling in Reducing Pain Associated With Non-invasive Treatments of Neurofibromatosis Type 1 Cutaneous Neurofibromas
NCT04054011	EARLY_PHASE1	COMPLETED	Safety and Efficacy of Deoxycholic Acid Injection for Reduction of Upper Inner Thigh Fat
NCT00757081	Not specified	COMPLETED	Study to Evaluate the Effect of Injection Lypolysis (Lipodissolve) Treatments to Reduce Body Fat
NCT00851747	Not specified	TERMINATED	A Study of the Efficacy of Subcutaneous Phosphatidylcholine and Deoxycholate Injections for Localized Fat Removal
NCT02438813	Not specified	COMPLETED	Condition of Submental Fullness and Treatment Outcomes Registry
NCT03284034	Not specified	TERMINATED	Cryolipolysis Versus ATX-101 (Deoxycholic Acid) for Upper Back Fat

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

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.

66 molecules share ≥1 primary target. Top 60 by shared-target count:

Molecule	Source	Status	Shared targets
chenodiol	ChEMBL + PubChem	Phase 4 (approved)	FPR1, GPBAR1, NR1H4
cyclosporine	ChEMBL + PubChem	Phase 4 (approved)	FPR1, NR1H4
CHOLIC ACID	ChEMBL	Phase 4 (approved)	GPBAR1, NR1H4
OBETICHOLIC ACID	ChEMBL	Phase 4 (approved)	GPBAR1, NR1H4
TAURURSODIOL	ChEMBL	Phase 4 (approved)	GPBAR1, NR1H4
TERN-101	ChEMBL	Phase 2	GPBAR1, NR1H4
FULVESTRANT	ChEMBL + PubChem	Phase 4 (approved)	NR1H4
REGORAFENIB	ChEMBL + PubChem	Phase 4 (approved)	NR1H4
ACETAMINOPHEN	ChEMBL	Phase 4 (approved)	NR1H4
APREPITANT	ChEMBL	Phase 4 (approved)	FPR1
ATORVASTATIN	ChEMBL	Phase 4 (approved)	NR1H4
BENZBROMARONE	ChEMBL	Phase 4 (approved)	NR1H4
CINACALCET	ChEMBL	Phase 4 (approved)	FPR1
CLOFAZIMINE	ChEMBL	Phase 4 (approved)	NR1H4
CLOTRIMAZOLE	ChEMBL	Phase 4 (approved)	NR1H4
DICLOFENAC	ChEMBL	Phase 4 (approved)	NR1H4
EPALRESTAT	ChEMBL	Phase 4 (approved)	NR1H4
ESTRADIOL	ChEMBL	Phase 4 (approved)	GPBAR1
FELODIPINE	ChEMBL	Phase 4 (approved)	NR1H4
FLUTRIMAZOLE	ChEMBL	Phase 4 (approved)	NR1H4
IVERMECTIN	ChEMBL	Phase 4 (approved)	NR1H4
KETOCONAZOLE	ChEMBL	Phase 4 (approved)	NR1H4
LEVOTHYROXINE	ChEMBL	Phase 4 (approved)	NR1H4
LOPERAMIDE	ChEMBL	Phase 4 (approved)	FPR1
LORATADINE	ChEMBL	Phase 4 (approved)	NR1H4
MONTELUKAST	ChEMBL	Phase 4 (approved)	FPR1
NIMODIPINE	ChEMBL	Phase 4 (approved)	NR1H4
ODEVIXIBAT	ChEMBL	Phase 4 (approved)	NR1H4
PENICILLIN G	ChEMBL	Phase 4 (approved)	FPR1
PERPHENAZINE	ChEMBL	Phase 4 (approved)	FPR1
PHENYLBUTAZONE	ChEMBL	Phase 4 (approved)	FPR1
PRANLUKAST	ChEMBL	Phase 4 (approved)	NR1H4
PRASTERONE	ChEMBL	Phase 4 (approved)	GPBAR1
PREDNISOLONE	ChEMBL	Phase 4 (approved)	GPBAR1
PROGESTERONE	ChEMBL	Phase 4 (approved)	GPBAR1
RALOXIFENE	ChEMBL	Phase 4 (approved)	NR1H4
REPAGLINIDE	ChEMBL	Phase 4 (approved)	NR1H4
RIMONABANT	ChEMBL	Phase 4 (approved)	NR1H4
SIMVASTATIN	ChEMBL	Phase 4 (approved)	NR1H4
SULCONAZOLE	ChEMBL	Phase 4 (approved)	NR1H4
SULFINPYRAZONE	ChEMBL	Phase 4 (approved)	FPR1
SUNITINIB	ChEMBL	Phase 4 (approved)	NR1H4
TESTOSTERONE	ChEMBL	Phase 4 (approved)	GPBAR1
TROGLITAZONE	ChEMBL	Phase 4 (approved)	NR1H4
URSODIOL	ChEMBL	Phase 4 (approved)	GPBAR1
ZAFIRLUKAST	ChEMBL	Phase 4 (approved)	NR1H4
ANDROGRAPHOLIDE	ChEMBL	Phase 3	NR1H4
CILOFEXOR	ChEMBL	Phase 3	NR1H4
ELOBIXIBAT	ChEMBL	Phase 3	NR1H4
GLYCOCHOLIC ACID	ChEMBL	Phase 3	GPBAR1
NORUCHOLIC ACID	ChEMBL	Phase 3	GPBAR1
TAUROLITHOCHOLIC ACID	ChEMBL	Phase 3	GPBAR1
VIDOFLUDIMUS	ChEMBL	Phase 3	NR1H4
FORETINIB	ChEMBL	Phase 2	FPR1
NIDUFEXOR	ChEMBL	Phase 2	NR1H4
NIGULDIPINE	ChEMBL	Phase 2	FPR1
PX-102	ChEMBL	Phase 2	NR1H4
STANOLONE	ChEMBL	Phase 2	GPBAR1
TAURODEOXYCHOLIC ACID	ChEMBL	Phase 2	GPBAR1
TROPIFEXOR	ChEMBL	Phase 2	NR1H4