Folic Acid

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Also known as Acide foliqueAcido folicoAcidum folicumB03BB01Bio scienceFolacinFolateFoldineFolettesFoliaminFolic acid component of folvronFolic acid component of vitapedFolicareFolicetFolipacFolsanFolviteIncafolicLexpec

Summary

Folic Acid (CHEMBL1622) is an approved small molecule (ATC V04CX02) targeting SLC19A1 and SLC46A1; indicated across 72 conditions including iron deficiency anemia and chronic kidney disease.

At a glance

  • Status: Approved (max clinical phase 4)
  • Modality: Small molecule
  • ATC class: V04CX02 (+2 more)
  • Targets: 2 (SLC19A1, SLC46A1)
  • Indications: 72 conditions
  • Clinical trials: 285
  • Chemistry: 441.4 Da · C19H19N7O6

Identifiers

Drug identity and classification

FieldValue
ChEMBL IDCHEMBL1622
NameFolic Acid
TypeSmall molecule
Max phase4
FDA approvedyes
PubChem CID135398658
ChEBICHEBI:27470
ATCV04CX02, B03BB01, B03BB51
Molecular formulaC19H19N7O6
Molecular weight441.4
InChIKeyOVBPIULPVIDEAO-LBPRGKRZSA-N

SMILES: C1=CC(=CC=C1C(=O)N[C@@H](CCC(=O)O)C(=O)O)NCC2=CN=C3C(=N2)C(=O)NC(=N3)N

IUPAC name: (2S)-2-[[4-[(2-amino-4-oxo-3H-pteridin-6-yl)methylamino]benzoyl]amino]pentanedioic acid

ChEBI definition: An N-acyl-amino acid that is a form of the water-soluble vitamin B9. Its biologically active forms (tetrahydrofolate and others) are essential for nucleotide biosynthesis and homocysteine remethylation.

Pharmacological roles (ChEBI): nutrient.

Other ChEBI roles (chemical / environmental): human metabolite, mouse metabolite.

Also known as: Acide folique, Acido folico, Acidum folicum, B03BB01, Bio science, Folacin, Folate, Foldine, Folettes, Foliamin, Folic acid, Folic acid component of folvron

Parent form; salt/anhydrous children: CHEMBL2107429

Patent coverage: 199,776 distinct patent families (525,158 SureChEMBL compound mentions), from 8 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).

GeneTargetActionpAffinityCancer dependencyUniProt
SLC19A1Reduced folate transporter 131.4%P41440
SLC46A1Proton-coupled folate transporter2.7%Q96NT5

Broader ChEMBL bioactivity targets: 17 (assay-derived). Sample: Tyrosyl-DNA phosphodiesterase 1, Lysine-specific demethylase 4E, Fructose-bisphosphate aldolase, Ferritin light chain, 15-hydroxyprostaglandin dehydrogenase [NAD(+)], Dihydrofolate reductase, Aldo-keto reductase family 1 member B1, Thymidylate synthase, Thyrotropin receptor, Solute carrier organic anion transporter family member 1A3.

Bioactivity

ChEMBL activities: 6 potent at pChembl ≥ 5 of 21 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

TargetpChemblTypeValueUnitActivity ID
P0ABQ46.09Kd810nMCHEMBL_ACT_817143
P0ABQ45.36Kd4400nMCHEMBL_ACT_817144
P0ABQ45.32Kd4800nMCHEMBL_ACT_817145
TSHR5.1Potency7943nMCHEMBL_ACT_3918974
Q639215AC5010000nMCHEMBL_ACT_25174327
KDM4E5Potency10000nMCHEMBL_ACT_3714365

Target pathways

Aggregated over 2 target gene(s): SLC19A1, SLC46A1.

Top Reactome pathways

6 total, by targets touching each:

PathwayTargetsGenes
Metabolism of folate and pterines2SLC19A1, SLC46A1
Metabolism1SLC19A1
Metabolism of water-soluble vitamins and cofactors1SLC19A1
Metabolism of vitamins and cofactors1SLC19A1
Iron uptake and transport1SLC46A1
Heme signaling1SLC46A1

Dominant GO biological processes

GO termTargets
folic acid transport2
folic acid metabolic process2
methotrexate transport2
folate transmembrane transport2
folate import across plasma membrane2
xenobiotic transmembrane transport1
female pregnancy1
response to xenobiotic stimulus1
response to toxic substance1
obsolete organic anion transport1
cyclic-GMP-AMP transmembrane import across plasma membrane1
positive regulation of cGAS/STING signaling pathway1
transport across blood-brain barrier1
vitamin transmembrane transport1
vitamin transport1

Indications & clinical

Indications

72 indications (11 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).

IndicationTrial phaseMONDOEFO
iron deficiency anemia4MONDO:0001356HP:0001891
chronic kidney disease4MONDO:0005300EFO:0003884
anemia4MONDO:0002280EFO:0004272
osteoporosis4MONDO:0005298EFO:0003882
acne4MONDO:0011438EFO:0003894
depressive disorder4MONDO:0002050MONDO:0002050
type 2 diabetes mellitus4MONDO:0005148MONDO:0005148
epilepsy3MONDO:0005027EFO:0000474
heart disorder3MONDO:0005267EFO:0003777
rheumatoid arthritis3MONDO:0008383EFO:0000685
hypertensive disorder3MONDO:0005044EFO:0000537
malaria3MONDO:0005136EFO:0001068
non-small cell lung carcinoma3MONDO:0005233EFO:0003060
cleft lip3MONDO:0004747EFO:0003959
colorectal neoplasm3MONDO:0005335EFO:0004142
male infertility3MONDO:0005372EFO:0004248
cleft palate3MONDO:0016064HP:0000175
gingival overgrowth3MONDO:0002507MONDO:0002507
severe acute respiratory syndrome3MONDO:0005091MONDO:0100096
Alzheimer disease3MONDO:0004975MONDO:0004975
type 1 diabetes mellitus3MONDO:0005147MONDO:0005147
neural tube defect3MONDO:0018075MONDO:0018075
tuberculosis3MONDO:0018076MONDO:0018076
premature ejaculation3MONDO:0001780EFO:0803321
systemic lupus erythematosus3MONDO:0007915MONDO:0007915
HIV infectious disease2MONDO:0005109EFO:0000764
autism2MONDO:0005260EFO:0003758
habitual spontaneous abortion2MONDO:0006774EFO:1000954
peripheral T-cell lymphoma, not otherwise specified2MONDO:0004964EFO:0000211
diabetic kidney disease2MONDO:0005016EFO:0000401
infertility disorder2MONDO:0005047EFO:0000545
lymphoma2MONDO:0005062EFO:0000574
mesothelioma2MONDO:0005065EFO:0000588
heart failure2MONDO:0005252EFO:0003144
breast neoplasm2MONDO:0021100EFO:0003869
atherosclerosis2MONDO:0005311EFO:0003914
upper aerodigestive tract neoplasm2MONDO:0005398EFO:0004284
psychotic disorder2MONDO:0005485EFO:0005407
head and neck cancer2MONDO:0005627EFO:0006859
uveitis2MONDO:0020283EFO:1001231
heavy metal poisoning2MONDO:0023305EFO:1001518
breast carcinoma2MONDO:0004989EFO:0000305
myocardial infarction2MONDO:0005068EFO:0000612
myocardial ischemia2MONDO:0024644EFO:1001375
mucositis2MONDO:0020579EFO:1001898
lung neoplasm2MONDO:0021117MONDO:0008903
coronary artery disorder2MONDO:0005010EFO:0001645
rectal cancer2MONDO:0006519EFO:1000657
sickle cell disease2MONDO:0011382MONDO:0011382
Parkinson disease2MONDO:0005180MONDO:0005180
polycystic ovary syndrome1MONDO:0008487EFO:0000660
neoplasm1MONDO:0005070EFO:0000616
primary cutaneous T-cell non-Hodgkin lymphoma1MONDO:0000607EFO:0002913
anaplastic large cell lymphoma1MONDO:0020325EFO:0003032
Sezary syndrome1MONDO:0017844EFO:1000785
mycosis fungoides1MONDO:0009691EFO:1001051
peritoneal neoplasm1MONDO:0006901MONDO:0002087
fallopian tube neoplasm1MONDO:0021092MONDO:0002158
ovarian cancer1MONDO:0008170MONDO:0008170
pulmonary alveolar proteinosis1MONDO:0001437MONDO:0001437

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

Clinical trials

Total trials: 285.

Phase distribution

PhaseTrials
Not specified93
PHASE260
PHASE450
PHASE336
PHASE118
PHASE2/PHASE313
PHASE1/PHASE210
EARLY_PHASE15

Top trials by phase / activity

NCTPhaseStatusTitle
NCT04579848PHASE4ACTIVE_NOT_RECRUITINGMethotrexate in Erosive Inflammatory Hand Osteoarthritis
NCT04974138PHASE4RECRUITINGChina Stroke Primary Prevention Trial 2 for Participants With H-type Hypertension and MTHFR 677 CC/CT Genotype (CSPPT2-CC/CT)
NCT04974151PHASE4RECRUITINGChina Stroke Primary Prevention Trial 2 for Participants With Hypertension and MTHFR 677 TT Genotype
NCT06610266PHASE4RECRUITINGEfficacy and Safety of Weifuchun Tablet in Reversing Gastric Intestinal Metaplasia
NCT06943482PHASE4NOT_YET_RECRUITINGComparing Effectiveness of Steroids and Methotrexate in Treatment of Chronic Inflammatory Breast Disease
NCT07029698PHASE4RECRUITINGA Study to See if a Combination of Vitamins That is Injected Into a Muscle is as Good and Safe as a Vitamin That is Taken by Mouth
NCT00130065PHASE4COMPLETEDThe Effect of Folic Acid on Efficacy of Sulfadoxine-pyrimethamine in Pregnant Women in Western Kenya
NCT00164970PHASE4COMPLETEDCan Oral Vitamin B12 and Folate Supplementation Preserve Cognitive Function of Patients With Early Dementia?
NCT00226356PHASE4COMPLETEDNatural Supplements for Unipolar Depression
NCT00249288PHASE4COMPLETEDFolate Supplementation in Schizophrenia
NCT00317005PHASE4COMPLETEDUremic Hyperhomocysteinemia -A Folate Trial for Possible Prevention of Cardiovascular Events
NCT00462345PHASE4COMPLETEDA Study of MabThera (Rituximab) in Combination With Methotrexate in Patients With Rheumatoid Arthritis Who Have Had an Inadequate Response to Anti-TNF Therapies.
NCT00491907PHASE4TERMINATEDEffect of Folic Acid on Endothelial and Baroreceptor Function in Patients With Heart Failure
NCT00514410PHASE4COMPLETEDFolate Augmentation of Treatment - Evaluation for Depression: a Randomised Controlled Trial
NCT00570856PHASE4TERMINATEDTrial of Folic Acid Effect on Hcy and cIMT After Kidney Tx
NCT00611806PHASE4COMPLETEDEffectiveness of Vitamin Supplementation in Treating People With Residual Symptoms of Schizophrenia
NCT00794885PHASE4COMPLETEDChina Stroke Primary Prevention Trial
NCT01050556PHASE4COMPLETEDFolic Acid and Creatine as Therapeutic Approaches for Lowering Blood Arsenic
NCT01263080PHASE4COMPLETEDEvaluation of Mirtazapine and Folic Acid for Schizophrenia:
NCT01283971PHASE4TERMINATEDA Study of RoActemra/Actemra (Tocilizumab) Versus Adalimumab in Combination With Methotrexate (MTX) in Patients With Moderate to Severe Active Rheumatoid Arthritis And an Inadequate Response to Treatment With Only One Tumor Necrosis Factor (TNF)-Inhibitor
NCT01308255PHASE4COMPLETEDInfliximab as Induction Therapy in Early Rheumatoid Arthritis (IDEA)
NCT01493674PHASE4COMPLETEDFolic Acid Supplementation in Eating Disorder
NCT01511835PHASE4UNKNOWNEfficacy of Myo-inositol in Preventing Gestational Diabetes in High-risk Pregnant Women
NCT01514942PHASE4COMPLETEDMyo-inositol Versus D-chiro-inositol in the Treatment of Polycystic Ovary Syndrome and Insulin Resistance: Evaluation of Clinical, Metabolic, Endocrine and Ultrasound Parameters
NCT01540747PHASE4COMPLETEDRole of Melatonin Supplementation in Follicular Fluid of in Vitro Fertilization (IVF) Patients With Polycystic Ovarian Syndrome
NCT01560065PHASE4COMPLETEDEffect of Treatment With Myo-inositol on Human Semen Parameters in Patients Undergoing In Vitro Fertilization Cycles
NCT01583959PHASE4COMPLETEDStudy of Different Doses of Folic Acid in Combination With Methotrexate in Rheumatoid Arthritis
NCT01608347PHASE4COMPLETEDLow Molecular Weight Heparin in Recurrent Miscarriage With Negative Antiphospholipid Antibodies
NCT01608750PHASE4UNKNOWNThe Efficacy of Pantoprazole Treatment in Patients With Functional Dyspepsia
NCT01626443PHASE4COMPLETEDRole of Myo-inositol and D-chiro Inositol on the Ovaric and Metabolic Functions
NCT01733329PHASE4COMPLETEDBuccal Misoprostol During Cesarean Section for Preventing Postpartum Hemorrhage
NCT01766310PHASE4COMPLETEDEffect of Folic Acid Supplementation on Plasma Homocysteine Level in Obese Children
NCT01871740PHASE4WITHDRAWNCSPPT- Chronic Kidney Diseases Study
NCT02031289PHASE4COMPLETEDImpact of Preoperative Treatment of Anemia and Iron Deficiency in Cardiac Surgery on Outcome.
NCT02150291PHASE4UNKNOWNA Pilot Study to Assess the Efficacy and Safety of Folic Acid and/or Vitamin B Complex on Hepatitis C Infected Patients Treated With Pegylated Interferon and Ribavirin.
NCT02300948PHASE4COMPLETEDOptimizing Periconceptional and Prenatal Folic Acid Supplementation
NCT02344888PHASE4UNKNOWNAdding Prednisolone During Ovulation Induction With CC in Lean Women With CC Resistant PCOS
NCT02444013PHASE4UNKNOWNFolic Acid for Prevention of Contrast Induced Nephropathy
NCT02644499PHASE4COMPLETEDComparison of Combination Disease Modifying Antirheumatic Drugs With Methotrexate Therapy in Early Rheumatoid Arthritis
NCT03040219PHASE4UNKNOWNFolic Acid Supplementation as an Appetizer in Primary Malnourished Egyptian Infants and Children

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

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.

6 molecules share ≥1 primary target. Top 6 by shared-target count:

MoleculeSourceStatusShared targets
METHOTREXATEChEMBL + PubChemPhase 4 (approved)SLC19A1, SLC46A1
PEMETREXEDChEMBL + PubChemPhase 4 (approved)SLC19A1, SLC46A1
PRALATREXATEChEMBL + PubChemPhase 4 (approved)SLC19A1, SLC46A1
RALTITREXEDChEMBLPhase 4 (approved)SLC19A1, SLC46A1
IndomethacinPubChemApprovedSLC46A1
LeucovorinPubChemApprovedSLC19A1