Ambroxol

Summary

Ambroxol (CHEMBL153479) is a phase-3 clinical-stage small molecule (ATC R05CB06) targeting GBA1; indicated across 8 conditions including respiratory system disorder and pharyngitis.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Small molecule
• ATC class: R05CB06 (+1 more)
• Targets: 1 (GBA1)
• Indications: 8 conditions
• Clinical trials: 28
• Chemistry: 378.1 Da · C13H18Br2N2O

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL153479
Name	Ambroxol
Type	Small molecule
Max phase	3
FDA approved	no
PubChem CID	2132
ATC	R05CB06, R02AD05
Molecular formula	C13H18Br2N2O
Molecular weight	378.1
InChIKey	JBDGDEWWOUBZPM-UHFFFAOYSA-N

SMILES: C1CC(CCC1NCC2=C(C(=CC(=C2)Br)Br)N)O

IUPAC name: 4-[(2-amino-3,5-dibromophenyl)methylamino]cyclohexan-1-ol

Also known as: Ambroxol, Tabcin, SID29215331, SID50085991, ambroxol, AMBROXOL, ambroxol hydrochloride

Parent form; salt/anhydrous children: CHEMBL1514634

Patent coverage: 5,048 distinct patent families (12,659 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 12,426 (98%) 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
GBA1	glucosylceramidase beta	Binding		0.5%	P04062

Broader ChEMBL bioactivity targets: 8 (assay-derived). Sample: Prelamin-A/C, Histamine H2 receptor, Thromboxane A2 receptor, Lysosomal acid glucosylceramidase, Cannabinoid receptor 1, Sodium-dependent serotonin transporter, Voltage-gated inwardly rectifying potassium channel KCNH2, Cytochrome P450 2D6.

Bioactivity

ChEMBL activities: 7 potent at pChembl ≥ 5 of 12 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
CNR1	8.92	AC50	1.2	nM	CHEMBL_ACT_25116729
CYP2D6	6.25	IC50	557.4	nM	CHEMBL_ACT_7629553
SLC6A4	5.74	Ki	1802	nM	CHEMBL_ACT_7630920
SLC6A4	5.47	IC50	3391	nM	CHEMBL_ACT_7630919
GBA1	5.39	IC50	4100	nM	CHEMBL_ACT_12095476
HRH2	5.2	AC50	6361	nM	CHEMBL_ACT_25114336
SLC6A4	5.19	AC50	6500	nM	CHEMBL_ACT_25149891

Target pathways

Aggregated over 1 target gene(s): GBA1.

Top Reactome pathways

2 total, by targets touching each:

Pathway	Targets	Genes
Association of TriC/CCT with target proteins during biosynthesis	1	GBA1
Glycosphingolipid catabolism	1	GBA1

Dominant GO biological processes

GO term	Targets
mitophagy	1
glucosylceramide catabolic process	1
autophagy	1
immune response	1
lysosome organization	1
cholesterol metabolic process	1
determination of adult lifespan	1
glycolipid biosynthetic process	1
cellular response to starvation	1
response to pH	1
microglia differentiation	1
regulation of macroautophagy	1
antigen processing and presentation	1
lipid storage	1
cerebellar Purkinje cell layer formation	1

Indications & clinical

Indications

8 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
respiratory system disorder	3	MONDO:0005087	EFO:0000684
pharyngitis	3	MONDO:0002258	MONDO:0002258
Parkinson disease	3	MONDO:0005180	MONDO:0005180
dementia	2	MONDO:0001627	HP:0000726
Gaucher disease	2	MONDO:0018150	MONDO:0018150
amyotrophic lateral sclerosis	2	MONDO:0004976	MONDO:0004976
Lewy body dementia	1	MONDO:0007488	EFO:0006792

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

Clinical trials

Total trials: 28.

Phase distribution

Phase	Trials
PHASE2	8
PHASE3	6
PHASE1	5
PHASE1/PHASE2	4
Not specified	3
PHASE4	1
PHASE2/PHASE3	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05663905	PHASE4	COMPLETED	Efficacy of Intravenous Amboxol Hydrochloride as an Adjunct Therapy for Severe Pneumonia in Critically Ill Patients
NCT05778617	PHASE3	RECRUITING	Ambroxol to Slow Progression in Parkinson Disease
NCT00144274	PHASE3	COMPLETED	Ambroxol Lozenges 20mg in Relieving the Pain of Sore Throat in Adolescent Patients With Acute Viral Pharyngitis
NCT00148499	PHASE3	COMPLETED	Efficacy and Tolerability of Ambroxol Lozenge 20 mf in Relieving Pain of Sore Throat in Pat. With Acute/Viralpharyngitis
NCT00525044	PHASE3	COMPLETED	Double-blind Trial to Investigate Efficacy and Tolerance of Ambroxol Lozenges 20 mg in Sore Throat
NCT03583658	PHASE3	COMPLETED	Efficacy and Safety Study of New Ambroxol Hardboiled Lozenges in Acute Pharyngitis
NCT03843541	PHASE3	COMPLETED	A Clinical Trial to Compare the Efficacy and Safety of 1-week Treatment of Intravenous N-acetylcysteine (NAC) 600 mg Twice Daily, Ambroxol Hydrochloride 30 mg Twice Daily and Placebo as Expectorant Therapies in Adult Chinese Patients With Respiratory Tract Diseases and Abnormal Mucus Secretions
NCT05830396	PHASE2/PHASE3	UNKNOWN	GRoningen Early-PD Ambroxol Treatment
NCT04405596	PHASE1/PHASE2	NOT_YET_RECRUITING	Ambroxol as a Novel Disease Modifying Treatment for Lewy Body Dementia
NCT04588285	PHASE2	RECRUITING	Ambroxol in New and Early DLB, A Phase IIa Multicentre Randomized Controlled Double Blind Clinical Trial
NCT01361802	PHASE2	COMPLETED	Ambroxol Spray Sore Throat Study
NCT01463215	PHASE1/PHASE2	SUSPENDED	Clinical Trial of Ambroxol in Patients With Type I Gaucher Disease
NCT01713179	PHASE1/PHASE2	COMPLETED	Effects of Oral Mucolytics on Tear Film and Ocular Surface
NCT02914366	PHASE2	UNKNOWN	Ambroxol as a Treatment for Parkinson’s Disease Dementia
NCT02941822	PHASE2	COMPLETED	Ambroxol in Disease Modification in Parkinson Disease
NCT03415269	PHASE2	COMPLETED	A Study of the Effect of 20 mg Ambroxol Hydrochloride on Acute Cough.
NCT03950050	PHASE2	COMPLETED	Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy
NCT05287503	PHASE2	COMPLETED	Ambroxol as a Disease-modifying Treatment in GBA-PD
NCT05959850	PHASE2	UNKNOWN	A Double-blind Randomised, Placebo-controlled Clinical Trial to Test Ambroxol Treatment in ALS
NCT06193421	PHASE1/PHASE2	UNKNOWN	High-Dose Ambroxol in GBA1-Related Parkinson
NCT01573663	PHASE1	COMPLETED	A Drug-Drug Interaction Study of Ambroxol and Levodropropizine
NCT02194257	PHASE1	COMPLETED	Investigation of the Metabolism and Pharmacokinetics of Ambroxol in Healthy Male Volunteers
NCT02194270	PHASE1	COMPLETED	Relative Bioavailability of Ambroxol Hydrochloride of Soft Pastilles Compared to Ambroxol Hydrochloride Syrup in Healthy Male and Female Volunteers
NCT02194296	PHASE1	COMPLETED	Relative Bioavailability of Ambroxol Hydrochloride Lozenge in Comparison to Ambroxol Hydrochloride Syrup (Mucosolvan®) in Healthy Female and Male Volunteers
NCT02572609	PHASE1	COMPLETED	Relative Bioavailability Study Between Two Formulations Containing Ambroxol Hydrochloride
NCT05002192	Not specified	UNKNOWN	A Retrospective, Real-world Study of ELP Used in the Expectorant Treatment of Community-acquired Pneumonia
NCT05558878	Not specified	UNKNOWN	Effect of Ambroxol in Diabetic Peripheral Neuropathy
NCT07285369	Not specified	TERMINATED	High-Dose Ambroxol in Pediatric Type III Gaucher Disease (GD3)

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.

11 molecules share ≥1 primary target. Top 11 by shared-target count:

Molecule	Source	Status	Shared targets
MEXILETINE	ChEMBL + PubChem	Phase 4 (approved)	GBA1
MIGALASTAT	ChEMBL + PubChem	Phase 4 (approved)	GBA1
TAMOXIFEN	ChEMBL + PubChem	Phase 4 (approved)	GBA1
CHLORHEXIDINE	ChEMBL	Phase 4 (approved)	GBA1
GENTIAN VIOLET	ChEMBL	Phase 4 (approved)	GBA1
GLUCONOLACTONE	ChEMBL	Phase 4 (approved)	GBA1
MIGLITOL	ChEMBL	Phase 4 (approved)	GBA1
AFEGOSTAT	ChEMBL	Phase 2	GBA1
DUVOGLUSTAT	ChEMBL	Phase 2	GBA1
NIZUBAGLUSTAT	ChEMBL	Phase 2	GBA1
Diltiazem	PubChem	Approved	GBA1