Oxybate
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Also known as .gamma.-hydroxybutyrateGamma hydroxybutyrateGamma-hydroxybutyrategamma-Hydroxybutyric acidgama-Hydroxybutyric acidSODIUM GAMMA-HYDROXYBUTYRATESODIUM OXYBATEGamma hydroxybutyric acid
Summary
Oxybate (CHEMBL1342) is an approved small-molecule general anaesthetic targeting HCAR1.
At a glance
- Status: Approved (max clinical phase 4)
- Modality: Small molecule
- Targets: 1 (HCAR1)
- Clinical trials: 26
- Chemistry: 104.1 Da · C4H8O3
Identifiers
Drug identity and classification
| Field | Value |
|---|---|
| ChEMBL ID | CHEMBL1342 |
| Name | Oxybate |
| Type | Small molecule |
| Max phase | 4 |
| FDA approved | yes |
| PubChem CID | 10413 |
| ChEBI | CHEBI:30830 |
| Molecular formula | C4H8O3 |
| Molecular weight | 104.1 |
| InChIKey | SJZRECIVHVDYJC-UHFFFAOYSA-N |
SMILES: C(CC(=O)O)CO
IUPAC name: 4-hydroxybutanoic acid
ChEBI definition: A 4-hydroxy monocarboxylic acid that is butyric acid in which one of the hydrogens at position 4 is replaced by a hydroxy group.
Pharmacological roles (ChEBI): general anaesthetic, GHB receptor agonist, sedative, neurotoxin.
Also known as: .gamma.-hydroxybutyrate, Gamma hydroxybutyrate, Gamma-hydroxybutyrate, Oxybate, gamma-Hydroxybutyrate, gamma-Hydroxybutyric acid, gama-Hydroxybutyric acid, gamma-hydroxybutyrate, Gamma-Hydroxybutyric Acid, Gamma-hydroxybutyric acid, OXYBATE, SODIUM GAMMA-HYDROXYBUTYRATE
Parent form; salt/anhydrous children: CHEMBL1200682, CHEMBL4594393, CHEMBL4594394, CHEMBL4594395
Patent coverage: 36,092 distinct patent families (111,544 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 |
|---|---|---|---|---|---|
| GABAB receptor | Partial agonist | ||||
| HCAR1 | HCA1 receptor | Full agonist | 1.82 | 0.6% | Q9BXC0 |
Broader ChEMBL bioactivity targets: 3 (assay-derived). Sample: GABA-A receptor; anion channel, Calcium/calmodulin-dependent protein kinase type II subunit alpha, Calcium/calmodulin-dependent protein kinase type II subunit alpha.
Bioactivity
ChEMBL activities: 5 potent at pChembl ≥ 5 of 6 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):
| Target | pChembl | Type | Value | Unit | Activity ID |
|---|---|---|---|---|---|
| P11275 | 5.52 | Ki | 3000 | nM | CHEMBL_ACT_24671406 |
| P11275 | 5.52 | Ki | 3000 | nM | CHEMBL_ACT_29151424 |
| O09028 | 5.37 | Ki | 4300 | nM | CHEMBL_ACT_19485614 |
| P11275 | 5.37 | Ki | 4300 | nM | CHEMBL_ACT_24671374 |
| CAMK2A | 5.37 | Ki | 4300 | nM | CHEMBL_ACT_24921753 |
Target pathways
Aggregated over 1 target gene(s): HCAR1.
Top Reactome pathways
2 total, by targets touching each:
| Pathway | Targets | Genes |
|---|---|---|
| Hydroxycarboxylic acid-binding receptors | 1 | HCAR1 |
| G alpha (i) signalling events | 1 | HCAR1 |
Dominant GO biological processes
| GO term | Targets |
|---|---|
| G protein-coupled receptor signaling pathway | 1 |
| adenylate cyclase-inhibiting G protein-coupled receptor signaling pathway | 1 |
| negative regulation of lipid catabolic process | 1 |
| signal transduction | 1 |
Indications & clinical
Indications
0 indications (0 at ChEMBL trial phase 4).
Clinical trials
Total trials: 26.
Phase distribution
| Phase | Trials |
|---|---|
| PHASE2 | 8 |
| PHASE4 | 7 |
| PHASE3 | 3 |
| PHASE2/PHASE3 | 3 |
| PHASE1 | 2 |
| Not specified | 2 |
| PHASE1/PHASE2 | 1 |
Top trials by phase / activity
| NCT | Phase | Status | Title |
|---|---|---|---|
| NCT00498485 | PHASE4 | TERMINATED | Use of Xyrem to Improve Sleep in Chronic Fatigue Syndrome |
| NCT00506974 | PHASE4 | COMPLETED | Enhancing Slow Wave Sleep With Sodium Oxybate |
| NCT00706186 | PHASE4 | TERMINATED | Safety and Feasibility of Sodium Oxybate in Mild Alzheimer’s Disease Patients |
| NCT01584934 | PHASE4 | WITHDRAWN | Sodium Oxybate in Patients With Chronic Fatigue Syndrome. |
| NCT02055898 | PHASE4 | COMPLETED | SWS And Daytime Functioning in Chronic FatiguE Syndrome (SAFFE) |
| NCT04006925 | PHASE4 | COMPLETED | Treatment of REM Sleep Behavior Disorder (RBD) With Sodium Oxybate |
| NCT04648423 | PHASE4 | COMPLETED | Evaluation of the Efficacy of Sodium Oxybate in the Long-term Maintenance of Abstinence in Alcoholic Patients |
| NCT00049803 | PHASE3 | COMPLETED | Safety and Efficacy of Xyrem Oral Solution (Sodium Oxybate) Compared With Placebo in Narcoleptic Patients |
| NCT00514995 | PHASE2/PHASE3 | COMPLETED | Sodium Oxybate in the Treatment of Binge Eating Disorder |
| NCT00803023 | PHASE3 | COMPLETED | Safety and Tolerability Study Comparing Sodium Oxybate Given as an Oral Solution to a Single-blinded Combination of Oral Tablets Plus Oral Solution in Subjects With Fibromyalgia |
| NCT02637648 | PHASE3 | UNKNOWN | Sodium Oxybate in Patients With Episodic and Chronic Cluster Headache |
| NCT03292458 | PHASE2/PHASE3 | COMPLETED | Sodium Oxybate in Spasmodic Dysphonia and Voice Tremor |
| NCT03597555 | PHASE2/PHASE3 | COMPLETED | Sodium Oxybate in Idiopathic Hypersomnia |
| NCT00383643 | PHASE2 | COMPLETED | Xyrem(Sodium Oxybate) and Ambien(Zolpidem Tartrate) in the Treatment of Chronic Insomnia. |
| NCT00594256 | PHASE2 | COMPLETED | Sodium Oxybate in Schizophrenia With Insomnia |
| NCT00598078 | PHASE2 | COMPLETED | Multiple-dose,Double-blind,Placebo-controlled Study of Sodium Oxybate in Patients With Essential Tremor |
| NCT00641186 | PHASE2 | COMPLETED | Trial of Xyrem for Excessive Daytime Sleepiness and Sleep Disturbance in Parkinson’s Disease (PD) |
| NCT00744393 | PHASE2 | WITHDRAWN | The Effect of Sodium Oxybate on Sleep Architecture |
| NCT00931164 | PHASE1/PHASE2 | COMPLETED | Sodium Oxybate in Patients With Alternating Hemiplegia of Childhood (AHC-SO) |
| NCT01961297 | PHASE2 | COMPLETED | Voice Tremor in Spasmodic Dysphonia: Central Mechanisms and Treatment Response |
| NCT02111122 | PHASE2 | COMPLETED | Study of the Symptomatic Effects of Nocturnal Sodium Oxybate in Parkinson’s Disease |
| NCT04224246 | PHASE2 | COMPLETED | Impact of Gamma-OH on Sleep in ICU Patients |
| NCT07041203 | PHASE1 | NOT_YET_RECRUITING | Extended-release Sodium Oxybate (Lumryz) in Spasmodic Dysphonia and Voice Tremor |
| NCT02215499 | PHASE1 | COMPLETED | A Phase 1, Single Dose Study of JZP-386 to Evaluate Safety, Pharmacokinetics and Pharmacodynamics |
| NCT02063217 | Not specified | COMPLETED | Modulation of CSF Amyloid-beta Concentrations Via Behavioral Sleep Deprivation and Pharmacological Sleep Induction |
| NCT04508166 | Not specified | COMPLETED | Towards a Post-exposition Pharmacological Prophylaxis for Post-traumatic Stress Disorder |
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.
2 molecules share ≥1 primary target. Top 2 by shared-target count:
| Molecule | Source | Status | Shared targets |
|---|---|---|---|
| lactic acid, l- | PubChem | Approved | HCAR1 |
| niacin | PubChem | Approved | HCAR1 |