Nitrofurantoin

Summary

Nitrofurantoin (CHEMBL572) is an approved small-molecule hepatotoxic agent (ATC J01XE01) targeting MPC2 and MPC1L; indicated across 4 conditions including urinary tract infection and cystitis.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: J01XE01 (+1 more)
• Targets: 2 (MPC2, MPC1L)
• Indications: 4 conditions
• Clinical trials: 40
• Chemistry: 238.16 Da · C8H6N4O5

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL572
Name	Nitrofurantoin
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	6604200
ChEBI	CHEBI:71415
ATC	J01XE01, J01XE51
Molecular formula	C8H6N4O5
Molecular weight	238.16
InChIKey	NXFQHRVNIOXGAQ-YCRREMRBSA-N

SMILES: C1C(=O)NC(=O)N1/N=C/C2=CC=C(O2)[N+](=O)[O-]

IUPAC name: 1-[(E)-(5-nitrofuran-2-yl)methylideneamino]imidazolidine-2,4-dione

ChEBI definition: An imidazolidine-2,4-dione that is hydantoin substituted at position 1 by a [(5-nitro-2-furyl)methylene]amino group. An antibiotic that damages bacterial DNA.

Pharmacological roles (ChEBI): hepatotoxic agent, antiinfective agent, antibacterial drug.

Also known as: Berkfurin, Ceduran, Dantafur, Furadantin, Furalan, Genfura, Macrobid, Macrodantin, Nitrofurantoin, Nitrofurantoin anhydrous, Nitrofurantoin component of macrobid, macrocrystalline

Parent form; salt/anhydrous children: CHEMBL1201000

Patent coverage: 7,611 distinct patent families (26,231 SureChEMBL compound mentions), from 2 matched compound structure(s). One matched structure accounts for 25,966 (99%) 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
MPC2	mitochondrial pyruvate carrier 2	Inhibition	5.48	1%	O95563
MPC1L	mitochondrial pyruvate carrier 1 like	Inhibition	5.48	0%	P0DKB6

Broader ChEMBL bioactivity targets: 16 (assay-derived). Sample: Tyrosyl-DNA phosphodiesterase 1, Microtubule-associated protein tau, Lysine-specific demethylase 4E, Streptokinase A, Survival motor neuron protein, Fructose-bisphosphate aldolase, Prelamin-A/C, 4’-phosphopantetheinyl transferase ffp, ATP-binding cassette sub-family C member 4, CDGSH iron-sulfur domain-containing protein 1.

Bioactivity

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

Target	pChembl	Type	Value	Unit	Activity ID
CISD1	6.09	Ki	816	nM	CHEMBL_ACT_16766193
LMNA	6	Potency	1000	nM	CHEMBL_ACT_3649975
P15917	5.9	Potency	1259	nM	CHEMBL_ACT_4633852
TP53	5.8	IC50	1580	nM	CHEMBL_ACT_25693991
P10520	5.24	EC50	5825	nM	CHEMBL_ACT_4941428
TDP1	5.05	Potency	8912	nM	CHEMBL_ACT_3928550

Target pathways

Aggregated over 2 target gene(s): MPC2, MPC1L.

Top Reactome pathways

3 total, by targets touching each:

Pathway	Targets	Genes
Aerobic respiration and respiratory electron transport	2	MPC1L, MPC2
Metabolism	2	MPC1L, MPC2
Pyruvate metabolism	2	MPC1L, MPC2

Dominant GO biological processes

GO term	Targets
pyruvate import into mitochondria	2
pyruvate decarboxylation to acetyl-CoA	1
positive regulation of insulin secretion involved in cellular response to glucose stimulus	1

Indications & clinical

Indications

4 indications (3 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
urinary tract infection	4	MONDO:0100338	EFO:0003103
cystitis	4	MONDO:0006032	EFO:1000025
bacterial infectious disease	4	MONDO:0005113	EFO:0000771
osteomyelitis	0	MONDO:0005246	EFO:0003102

Clinical trials

Total trials: 40.

Phase distribution

Phase	Trials
PHASE4	22
PHASE3	6
Not specified	5
EARLY_PHASE1	3
PHASE1	2
PHASE2	1
PHASE1/PHASE2	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05519072	PHASE4	RECRUITING	Use of Prophylactic Antibiotics Prior to OnabotulinumtoxinA
NCT05652374	PHASE4	RECRUITING	Gag Therapy for Recurrent Urinary Tract Infection Assessing Comparability to International Nitrofurantoin Gold Standard Study
NCT06387329	PHASE4	RECRUITING	Bladder Botox UTI Antibiotic Prophylaxis
NCT06518291	PHASE4	NOT_YET_RECRUITING	COMPARISON OF NITROFURANTOIN WITH FOSFOMYCIN in TREATING CYSTITIS IN WOMEN
NCT06709196	PHASE4	RECRUITING	Clinical Trial Testing Whether Targeted Antibiotic Prophylaxis Can Reduce Infections After Cystectomy Compared to Empiric Prophylaxis
NCT07186465	PHASE4	RECRUITING	Microbiome-Modulating Prophylaxis RCT: Antibiotic vs Gynoflor in Postmenopausal Women With Recurrent Cystitis
NCT00361998	PHASE4	WITHDRAWN	Nitrofurantoin Macrocrystals 3 Days Versus 7 Days in the Treatment of Women With Uncomplicated Cystitis
NCT00734968	PHASE4	COMPLETED	Does Treatment With Macrobid Reduce Urinary Tract Infections in Patients Receiving a Sub-Urethral Sling for Incontinence
NCT01092351	PHASE4	COMPLETED	Efficacy and Safety of Nitrofurantoin in the Treatment of Uncomplicated Urinary Tract Infections in Adults
NCT01450800	PHASE4	COMPLETED	Suppressive Therapy With Oral Antibiotics for Prevention of Postoperative Urinary Tract Infection (UTI)
NCT01966653	PHASE4	UNKNOWN	Study Comparing Nitrofurantoin to Fosfomycin for Acute Urinary Tract Infection in Women
NCT02375295	PHASE4	UNKNOWN	Struvite Stones Antibiotic Study
NCT02727322	PHASE4	COMPLETED	Nitrofurantoin Prophylaxis During Catheter-managed Acute Urinary Retention After Pelvic Reconstructive Surgery
NCT03287089	PHASE4	COMPLETED	Nitrofurantoin Administration for the Prevention of Short-Term Catheter Associated Urinary Tract Infection After Pelvic Surgery
NCT03299387	PHASE4	WITHDRAWN	INtravesical Antimicrobial Agents v STANDard Oral Antibiotics for the Treatment of Acute UTI in Women With rUTI
NCT03716804	PHASE4	COMPLETED	Establish the Relationship Between Shift in Prescribing Pattern and Associated Shift in Sensitivity Pattern of Causative Microbes in UTI Patients in a Closed Community
NCT04502095	PHASE4	COMPLETED	Prophylactic Antibiotics for Urinary Tract Infections After Robot-Assisted Radical Cystectomy
NCT04900571	PHASE4	COMPLETED	Comparison of Nitrofurantoin and Calcium Hydroxide as Intracanal Medicaments in Alleviating Post-operative Pain
NCT04959331	PHASE4	COMPLETED	Clinical Effectiveness and Bacteriological Eradication of 4 Short-course Antibiotics for Uncomplicated UTIs in Women.
NCT05274672	PHASE4	WITHDRAWN	Role of Prophylactic Postoperative Antibiotics in HoLEP
NCT05600023	PHASE4	COMPLETED	The Effectiveness of Single Antibiotic Paste Nitrofurantoin Versus Double Antibiotic Paste
NCT06332781	PHASE4	COMPLETED	Intravesical Gentamicin to Prevent Recurrent UTI
NCT06815549	PHASE3	NOT_YET_RECRUITING	An Open Clinical Trial of the Safety and Efficacy of the Drug Fluorothiazinone, 300 Mg Tablets with the Participation of Adult Patients with Chronic Bacterial Cystitis
NCT02021006	PHASE3	UNKNOWN	Antibiotic Prophylaxis and Renal Damage In Congenital Abnormalities of the Kidney and Urinary Tract
NCT04020341	PHASE3	COMPLETED	A Study to Evaluate Efficacy and Safety of Gepotidacin in the Treatment of Uncomplicated Urinary Tract Infection (UTI)
NCT04187144	PHASE3	COMPLETED	Comparative Study to Evaluate Efficacy and Safety of Gepotidacin to Nitrofurantoin in Treatment of Uncomplicated Urinary Tract Infection (UTI)
NCT05254808	PHASE3	TERMINATED	EXtended Use of FOsfomycin for the Treatment of CYstitis in Primary Care
NCT05630833	PHASE3	COMPLETED	A Study to Investigate the Efficacy and Safety With Gepotidacin in Japanese Female Participants With Uncomplicated Urinary Tract Infection (Acute Cystitis)
NCT07198919	PHASE1/PHASE2	ENROLLING_BY_INVITATION	Comparison of Inter-appointment Pain Reduction During Root Canal Treatment in Symptomatic Apical Periodontitis by Using Intracanal Medicaments; Chlorhexidine Gel and Single Antibiotic Paste Nitrofurantoin
NCT03425396	PHASE2	COMPLETED	Oral Omadacycline vs. Oral Nitrofurantoin for the Treatment of Cystitis
NCT06907199	PHASE1	RECRUITING	Daily Nitrofurantoin Versus Bladder Fulguration Plus Daily Nitrofurantoin for Women With Recurrent Urinary Tract Infections
NCT05138822	PHASE1	COMPLETED	Safety, Tolerability, Pharmacokinetic and Microbiological Investigation of GSK3882347 in Female Participants With Urinary Tract Infections
NCT07603934	EARLY_PHASE1	RECRUITING	The Effect of Different Intra-canal Medications on Postoperative Pain
NCT02789579	EARLY_PHASE1	UNKNOWN	The Preventive Infection Role of One Week Antibiotics Before Minimally Invasive Upper Tract Lithotomy
NCT05074628	EARLY_PHASE1	UNKNOWN	Effect of Nitrofurantion Used as an Intracanal Medicament
NCT07202832	Not specified	NOT_YET_RECRUITING	Urine Alkalinization for UTI in Women: RCT
NCT00678041	Not specified	TERMINATED	Nitrofurantoin and Urinary Tract Infections (UTIs)
NCT02092558	Not specified	COMPLETED	Evaluation of Immunological Disorders of T Lymphocytes and Endocrinological Disorders as Pathogen Factors in Patients With Metaplasia of Urinary Bladder
NCT02829060	Not specified	COMPLETED	Endourology Disease Group Excellence (EDGE) Consortium: Antibiotics (Abx) for Percutaneous Nephrolithotomy (PCNL) Part 2
NCT02911662	Not specified	TERMINATED	Treatment of Asymptomatic Bacteriuria in Pregnancy

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.

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

Molecule	Source	Status	Shared targets
MITOGLITAZONE	ChEMBL	Phase 2	MPC2