Voriconazole

Summary

Voriconazole (CHEMBL638) is an approved small-molecule P450 inhibitor (ATC J02AC03); indicated across 22 conditions including aspergillosis and candidemia.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: J02AC03
• Indications: 22 conditions
• Clinical trials: 106
• Chemistry: 349.31 Da · C16H14F3N5O

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL638
Name	Voriconazole
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	71616
ChEBI	CHEBI:10023
ATC	J02AC03
Molecular formula	C16H14F3N5O
Molecular weight	349.31
InChIKey	BCEHBSKCWLPMDN-MGPLVRAMSA-N

SMILES: C[C@@H](C1=NC=NC=C1F)[C@](CN2C=NC=N2)(C3=C(C=C(C=C3)F)F)O

IUPAC name: (2R,3S)-2-(2,4-difluorophenyl)-3-(5-fluoropyrimidin-4-yl)-1-(1,2,4-triazol-1-yl)butan-2-ol

ChEBI definition: A triazole-based antifungal agent used for the treatment of esophageal candidiasis, invasive pulmonary aspergillosis, and serious fungal infections caused by Scedosporium apiospermum and Fusarium spp. It is an inhibitor of cytochrome P450 2C9 (CYP2C9) and CYP3A4.

Pharmacological roles (ChEBI): P450 inhibitor.

Also known as: NSC-759888, UK-109,496, UK-109496, Vfend, Voriconazol, Voriconazole, Voriconazole accord, voriconazole, SID26719857, VORICONAZOLE, SID144205794, SID170464890

Patent coverage: 6,607 distinct patent families (23,088 SureChEMBL compound mentions), from 3 matched compound structure(s). One matched structure accounts for 22,836 (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

Broader ChEMBL bioactivity targets: 11 (assay-derived). Sample: Sterol 14-alpha demethylase cyp51A, Lanosterol 14-alpha demethylase, Cytochrome P450 3A, Lanosterol 14-alpha demethylase, Cytochrome P450 2C9, Cytochrome P450 3A4, Cytochrome P450 2C19, Mitogen-activated protein kinase 1, Cholesterol 24-hydroxylase, Cytochrome P450 2B6, Cholesterol side-chain cleavage enzyme, mitochondrial.

Bioactivity

ChEMBL activities: 25 potent at pChembl ≥ 5 of 39 total. Top 100 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
CYP46A1	7.96	Ki	11	nM	CHEMBL_ACT_19320617
CYP46A1	7.66	IC50	22	nM	CHEMBL_ACT_19320597
CYP46A1	7.33	IC50	47	nM	CHEMBL_ACT_19320598
CYP46A1	7.3	Kd	50	nM	CHEMBL_ACT_19320549
B8NUK6	7.25	Kd	56	nM	CHEMBL_ACT_18303837
P10613	6.78	Kd	165	nM	CHEMBL_ACT_18303829
CYP2B6	6.47	Ki	340	nM	CHEMBL_ACT_5089030
CYP2B6	6.4	Ki	400	nM	CHEMBL_ACT_5089029
P10613	6.19	IC50	650	nM	CHEMBL_ACT_26016124
CYP3A4	6.18	Ki	660	nM	CHEMBL_ACT_5089034
CYP2B6	6.1	IC50	790	nM	CHEMBL_ACT_5089021
CYP2B6	5.92	IC50	1190	nM	CHEMBL_ACT_5089020
CYP2B6	5.77	IC50	1710	nM	CHEMBL_ACT_5089006
CYP2B6	5.72	IC50	1900	nM	CHEMBL_ACT_5089024
CYP2C9	5.55	Ki	2800	nM	CHEMBL_ACT_5089031
CYP3A4	5.54	IC50	2900	nM	CHEMBL_ACT_5089009
CYP3A4	5.53	Ki	2970	nM	CHEMBL_ACT_5089033
CYP2C9	5.44	IC50	3620	nM	CHEMBL_ACT_5089007
Q4WNT5	5.44	Kd	3630	nM	CHEMBL_ACT_5229871
CYP2B6	5.43	IC50	3700	nM	CHEMBL_ACT_5089023
P00189	5.32	Kd	4800	nM	CHEMBL_ACT_19320594
CYP2C19	5.29	Ki	5100	nM	CHEMBL_ACT_5089032
CYP2C19	5.28	IC50	5250	nM	CHEMBL_ACT_5089008
CYP2C9	5.08	IC50	8400	nM	CHEMBL_ACT_5089010
CYP2C19	5.06	IC50	8700	nM	CHEMBL_ACT_5089011

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

5 approved indications. FDA phase 4, plus an anticancer drug’s labelled cancer uses (which ChEMBL often logs at phase 3).

Indication	Phase	MONDO	EFO
aspergillosis	4	MONDO:0005657	EFO:0007157
candidemia	4	MONDO:0044070	EFO:1001282
candidiasis	4	MONDO:0002026	MONDO:0002026
fungal infectious disease	4	MONDO:0002041	MONDO:0002041
fusariosis	4	MONDO:0016426	EFO:1001795

14 diseases in clinical trials (phase 1–3, investigational — not approved indications). Highest ChEMBL trial phase per disease; a non-cancer approved use is occasionally logged at phase 3 here.

Disease (in trials)	Phase	MONDO	EFO
acute myeloid leukemia	3	MONDO:0018874	EFO:0000222
leukemia	3	MONDO:0005059	EFO:0000565
candidiasis, invasive	3	MONDO:0044067	EFO:1001283
neuroaspergillosis	3	MONDO:0005873	EFO:0007393
leishmaniasis	3	MONDO:0011989	EFO:0005044
myelodysplastic syndrome	2	MONDO:0018881	EFO:0000198
allergic bronchopulmonary aspergillosis	2	MONDO:0015243	EFO:0007140
HIV infectious disease	1	MONDO:0005109	EFO:0000764
B-cell chronic lymphocytic leukemia	1	MONDO:0004948	EFO:0000095
neutropenia	1	MONDO:0001475	MONDO:0001475
graft versus host disease	1	MONDO:0013730	MONDO:0013730
lymphoid neoplasm	1	MONDO:0005157	EFO:0001642
asthma	1	MONDO:0004979	MONDO:0004979
neoplasm	1	MONDO:0005070	MONDO:0004992

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

Clinical trials

Total trials: 106.

Phase distribution

Phase	Trials
PHASE3	28
PHASE1	23
Not specified	22
PHASE4	14
PHASE2	12
PHASE2/PHASE3	3
PHASE1/PHASE2	3
EARLY_PHASE1	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00143312	PHASE4	COMPLETED	Voriconazole for Secondary Prophylaxis of Invasive Fungal Infections in Patients With Allogeneic Stem Cell Transplants
NCT00288197	PHASE4	COMPLETED	A Study To Assess The Efficacy And Safety Of Voriconazole In Chinese Patients With Serious Deep Tissue Fungal Infections
NCT00300677	PHASE4	COMPLETED	To Determine The Amount Of Voriconazole In The Brain After 2 Loading Doses And 3 Maintenance Doses Over 3 Days
NCT00423163	PHASE4	WITHDRAWN	A Study to Evaluate the Effectiveness of Voriconazole + Micafungin Versus Voriconazole Alone for Invasive Aspergillosis
NCT00455364	PHASE4	TERMINATED	Voriconazole as Prophylactic Therapy in Lung Transplant Recipients
NCT00496197	PHASE4	COMPLETED	Study Will Evaluate The Safety And Efficacy Of Anidulafungin In Patients With Candidemia Or Invasive Candidiasis
NCT00548262	PHASE4	COMPLETED	This Is An Open-Label, Non-Comparative Study Designed To Evaluate A Short Course Of IV Anidulafungin, Followed Optionally By Oral Voriconazole, For The Treatment Of Candidemia And Invasive Candidiasis
NCT00620074	PHASE4	TERMINATED	Study to Test the Combination of Voriconazole and Anidulafungin in Patients Who Have, or Are Thought to Have, Invasive Aspergillosis and Who Are Unable to Take a Common Antifungal Therapy (Polyene)
NCT00647907	PHASE4	COMPLETED	A Study of the Efficacy and Safety of Voriconazole for the Treatment of Fungal Infections
NCT00940017	PHASE4	COMPLETED	A Study To Assess The Anidulafungin And Voriconazole Concentration In Lung Following Intravenous Administration In Healthy Subjects
NCT01539330	PHASE4	COMPLETED	Assessment of Target Site Pharmacokinetics of Voriconazole in Healthy Volunteers During Sequence Therapy
NCT02234739	PHASE4	UNKNOWN	Voriconazole for IPA in Chinese Patients With COPD
NCT02283905	PHASE4	TERMINATED	Amphotericin-B and Voriconazole for Pulmonary Blastomycosis
NCT02576366	PHASE4	COMPLETED	Phenotypic Drug Probes as Predictors of Drug-drug Interactions With Tacrolimus
NCT00001646	PHASE3	COMPLETED	Voriconazole vs. Amphotericin B in the Treatment of Invasive Aspergillosis
NCT00001757	PHASE3	COMPLETED	An Open Label, Non-Comparative, Multicenter, Phase III Trial of the Efficacy, Safety and Toleration of Voriconazole in the Primary or Secondary Treatment of Invasive Fungal Infections
NCT00001810	PHASE3	COMPLETED	An Open Label, Non-Comparative, Multicenter, Phase III Trial of the Efficacy, Safety and Toleration of Voriconazole in the Primary or Secondary Treatment of Invasive Fungal Infections
NCT00001940	PHASE3	COMPLETED	Voriconazole to Treat Fungal Infections
NCT00003031	PHASE3	COMPLETED	Comparison of Voriconazole and Amphotericin B in Treating Patients With Aspergillosis
NCT00015665	PHASE3	COMPLETED	Emergency Use of Voriconazole in Patients With Life-Threatening Invasive Fungal Infections
NCT00075803	PHASE3	COMPLETED	Comparison of Fluconazole vs Voriconazole to Treat Fungal Infections for Blood and Marrow Transplants (BMT CTN 0101)
NCT00150345	PHASE3	COMPLETED	Immediate vs. Deferred Empirical Antifungal Treatment With Voriconazole In Neutropenic Patients
NCT00152594	PHASE3	TERMINATED	Voriconazole or Placebo in the Prophylaxis of Lung Infiltrates in Patients Undergoing Induction Chemotherapy for Acute Myelogenous Leukemia
NCT00159822	PHASE2/PHASE3	COMPLETED	Voriconazole For Chronic Bronchopulmonary Aspergillosis
NCT00412893	PHASE3	COMPLETED	Isavuconazole (BAL8557) for Primary Treatment of Invasive Aspergillosis
NCT00413218	PHASE3	COMPLETED	Isavuconazole (BAL8557) in the Treatment of Candidemia and Other Invasive Candida Infections
NCT00531479	PHASE3	COMPLETED	Anidulafungin Plus Voriconazole Versus Voriconazole For The Treatment Of Invasive Aspergillosis
NCT00624143	PHASE3	UNKNOWN	Antifungal Prophylaxis in Pediatric Acute Leukemia
NCT00689338	PHASE3	COMPLETED	Anidulafungin Candidemia/Invasive Candidiasis Intensive Care Study
NCT00836875	PHASE3	TERMINATED	A Study To Evaluate The Safety Of Voriconazole As Treatment Of Invasive Aspergillosis (Fungal Infection) And Other Rare Molds In Children
NCT00893555	PHASE3	COMPLETED	Pharmacologic Optimization of Voriconazole
NCT00904995	PHASE3	COMPLETED	Beta Glucan Assay in Patients Receiving Voriconazole Prophylaxis
NCT00996736	PHASE3	COMPLETED	Mycotic Ulcer Treatment Trial I
NCT00997035	PHASE3	COMPLETED	The Mycotic Ulcer Treatment Trial II: A Randomized Trial Comparing Oral Voriconazole vs Placebo
NCT01092832	PHASE3	TERMINATED	A Study Of The Safety, Tolerability And Effective Of Voriconazole For The Treatment Of Serious Candida Infection And Candida Infection Of The Throat In Pediatric Patients
NCT01188759	PHASE3	WITHDRAWN	Voriconazole And Anidulafungin Combination For Invasive Aspergillosis In Pediatric Subjects
NCT01503515	PHASE3	COMPLETED	Caspofungin Acetate, Fluconazole, or Voriconazole in Preventing Fungal Infections in Patients Following Donor Stem Cell Transplant
NCT01621321	PHASE2/PHASE3	COMPLETED	Voriconazole Versus Oral Steroids in Allergic Bronchopulmonary Aspergillosis
NCT01782131	PHASE3	COMPLETED	A Study of the Safety and Efficacy of Posaconazole Versus Voriconazole for the Treatment of Invasive Aspergillosis (MK-5592-069)
NCT02180165	PHASE3	COMPLETED	Assessing the Safety and Efficacy of MK-5592 (Posaconazole) in Japanese Participants With Fungal Infection (MK-5592-101)
NCT02731638	PHASE3	COMPLETED	Mycotic Antimicrobial Localized Injection for Treatment of Corneal Ulcers
NCT03230058	PHASE2/PHASE3	UNKNOWN	Combination Treatment of 5% Natamycin and 1% Voriconazole in Fungal Keratitis
NCT03471988	PHASE3	COMPLETED	Clinical Study of AK1820 (Isavuconazonium Sulfate) for the Treatment of Deep Mycosis
NCT04824417	PHASE3	COMPLETED	A Randomized Controlled Trial to Compare the Clinical Outcomes With Six Months of Therapy With Oral Itraconazole Versus Oral Voriconazole for Management of Treatment naïve Subjects With Chronic Pulmonary Aspergillosis
NCT05708625	PHASE3	UNKNOWN	Intralesional Voriconazole, or Intralesional Cryotherapy, or Oral Doxycycline in the Treatment of Cutaneous Leishmaniasis
NCT00059878	PHASE2	COMPLETED	Voriconazole With or Without Interferon Gamma in Treating Patients With Aspergillosis or Other Fungal Infections
NCT00066599	PHASE2	COMPLETED	Voriconazole in Preventing Fungal Infections in Children With Neutropenia After Chemotherapy
NCT00174473	PHASE1/PHASE2	COMPLETED	A Study to Investigate the Pharmacokinetics, Safety and Tolerability of Voriconazole in Children
NCT00238355	PHASE2	TERMINATED	Voriconazole and Caspofungin Acetate in Treating Invasive Fungal Infections in Patients With Weakened Immune Systems
NCT00418951	PHASE2	COMPLETED	Liposomal Amphotericin B (Ambisome) Versus Oral Voriconazole for the Prevention of Invasive Fungal Infections
NCT00556998	PHASE2	COMPLETED	A Multiple-Dose Study To Evaluate The Pharmacokinetics And Safety Of Voriconazole In Immunocompromised Adolescents
NCT00557362	PHASE1/PHASE2	COMPLETED	Therapeutic Exploratory Study of Comparing Natamycin and Voriconazole to Treat Fungal Corneal Ulcer
NCT00739934	PHASE2	COMPLETED	Study Of The Pharmacokinetics And Safety Of Voriconazole In Children 2 To 11 Years Old Who Are At High Risk For Systemic Fungal Infection
NCT01207128	PHASE2	WITHDRAWN	Trial of Combination Antifungal Therapy (Vori+Mica vs. Vori+Placebo) in Invasive Aspergillosis
NCT01383993	PHASE2	COMPLETED	Study Of The Pharmacokinetics And Safety Of Voriconazole In Children 2 To Less Than 15 Years Old Who Are At High Risk For Systemic Fungal Infection
NCT01576315	PHASE2	COMPLETED	ATCF (Azole Therapy in Cystic Fibrosis)
NCT01887457	PHASE2	SUSPENDED	Individualisation of Voriconazole Antifungal Therapy Antifungal Therapy
NCT02014558	PHASE1/PHASE2	COMPLETED	Dose Escalation Study Investigating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics of ASP2215 in Patients With Relapsed or Refractory Acute Myeloid Leukemia
NCT03672292	PHASE2	TERMINATED	Evaluate Safety and Efficacy of the Coadministration of Ibrexafungerp With Voriconazole in Patients With Invasive Pulmonary Aspergillosis
NCT04294641	PHASE2	COMPLETED	Front Line Ibrutinib Without Corticosteroids for Newly Diagnosed Chronic Graft-versus-Host Disease
NCT07426042	PHASE1	NOT_YET_RECRUITING	Effect of Dexamethasone on CYP Enzyme Activity in Healthy Male Subjects
NCT07457203	PHASE1	NOT_YET_RECRUITING	Study on the Drug Interaction Between Buagafuran and Voriconazole
NCT00005912	PHASE1	COMPLETED	Voriconazole to Prevent Systemic Fungal Infections in Children
NCT00136968	PHASE1	COMPLETED	Assessment of Voriconazole Penetration Into the Brain by Fluorine-Magnetic Resonance Spectroscopy
NCT00353158	PHASE1	COMPLETED	A Pilot of Pediatric/Adult Study of Gene Expression Profiling and Clinical Characterization of Phototoxicity
NCT00792246	PHASE1	COMPLETED	Voriconazole Pharmacokinetics in Children With Gastrointestinal Graft Versus Host Disease
NCT00833482	PHASE1	COMPLETED	Drug Interactions Between Voriconazole and Atazanavir Coadministered as Atazanavir/Ritonavir in Healthy Participants
NCT00855101	PHASE1	COMPLETED	A Pharmacokinetic And Safety Study Of Voriconazole In Healthy Male And Female Adults
NCT00942773	PHASE1	COMPLETED	Influence of CYP2C19 Genotype on the Pharmacokinetics (PK) of Voriconazole
NCT01080651	PHASE1	COMPLETED	Influence of Drug-drug Interactions on the Pharmacokinetics (PK) of Voriconazole
NCT01657201	PHASE1	COMPLETED	Study to Evaluate the Pharmacokinetic Properties of SYP-1018 (Voriconazole-loaded PNP) and Voriconazole (Vfend)
NCT01787032	PHASE1	COMPLETED	Drug-drug Interaction Trial of BI 113608 in Combination With Ketoconazole and Voriconazole in Healthy Male Subjects
NCT01950507	PHASE1	TERMINATED	Evaluation of Budesonide and How It Interacts With Antifungal Drugs in People With Gastrointestinal Graft-Versus-Host Disease
NCT02227797	PHASE1	COMPLETED	MT2013-37R: Voriconazole Monitoring in Pediatric Stem Cell Transplant Patients
NCT02381080	PHASE1	COMPLETED	Interaction Study of Ibrutinib and Cytochrome P450 (CYP) 3A Inhibitors in Participants With B-cell Malignancy
NCT03515382	PHASE1	COMPLETED	A Study in Healthy Male Volunteers to Look at How the Test Medicine GLPG1690 is Taken up by the Body When Doses of Itraconazole and Voriconazole Are Given to Healthy Volunteers
NCT04145583	PHASE1	COMPLETED	A Study Evaluating Drug-Drug Interaction (DDI) Between HSK3486 Injectable Emulsion and Voriconazole Tablets
NCT04229303	PHASE1	COMPLETED	Phase 1 Three Part SAD, MAD & Cross-Over Study of ZP-059 in Healthy and Asthmatic Subjects
NCT04551963	PHASE1	COMPLETED	Interaction Study of Zanubrutinib With Moderate and Strong CYP3A Inhibitors in Participants With B-Cell Malignancies
NCT04865419	PHASE1	TERMINATED	Study of AZD0466 Monotherapy or in Combination in Patients With Advanced Haematological Malignancies
NCT05264142	PHASE1	UNKNOWN	Risk of CYP2C19 Phenoconversion in Healthy Volunteers With Rapid, Normal, and Intermediate Predicted Metabolizers’ Status
NCT05458102	PHASE1	TERMINATED	Drug-Drug Interaction Study of Vesatolimod in Adults With HIV-1 Who Have Very Low or Undetectable Virus Levels
NCT06493409	PHASE1	COMPLETED	A Study to Assess the Effect of Voriconazole and Quinidine on the Pharmacokinetics of a Single Dose of Repotrectinib in Healthy Participants
NCT04072640	EARLY_PHASE1	UNKNOWN	Three Induction Treatments on Cryptococcal Meningitis
NCT00023530	Not specified	COMPLETED	Blood and Marrow Transplant Clinical Research Network
NCT00079222	Not specified	COMPLETED	Voriconazole Compared With Itraconazole in Preventing Fungal Infections in Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
NCT00721578	Not specified	COMPLETED	A Study Of Indian Patients Receiving Therapy For Systemic Fungal Infections
NCT01073631	Not specified	COMPLETED	Non-Interventional Post Marketing Surveillance Study To Observe The Safety And Efficacy Of Vfend® Tablet In Korea
NCT01101386	Not specified	COMPLETED	Study of Cyclodextrin (SBECD) and Voriconazole Blood Concentrations During Continuous Dialysis
NCT01104376	Not specified	COMPLETED	Determine the Effects of Gene Differences and Voriconazole on Enzyme CYP2B6 Activity in the Liver in Healthy Volunteers
NCT01137292	Not specified	COMPLETED	Open, Non Comparative Study Of Voriconazole In Slovak Patients With Very High Risk Of Developing An Invasive Fungal Infection
NCT01148160	Not specified	TERMINATED	Voriconazole Trough Plasma Levels : Genetic Polymorphism, Efficacy, Safety in Patients With Hematologic Malignancy
NCT01151085	Not specified	COMPLETED	Voriconazole (Vfend) Drug Use Investigation (Regulatory Post Marketing Commitment Plan)
NCT01344512	Not specified	COMPLETED	Population Pharmacokinetics of Anti-infectious Drugs in Children
NCT01480219	Not specified	COMPLETED	Evaluation Of The Importance Of Risk-Factor Adjustment For Assessing The Relationship Between Voriconazole Utilization And The Development Of Non-Melanoma Skin Cancer Among Lung And Heart/Lung Transplant Patients, 2002-2009g
NCT01660334	Not specified	COMPLETED	Voriconazole (Vfend) Special Investigation (Regulatory Post Marketing Commitment Plan)
NCT02100761	Not specified	UNKNOWN	CYP 2C19 Polymorphism and Voriconazole Trough Concentration in Chinese Adult Patients
NCT02396225	Not specified	TERMINATED	Concentrations of Voriconazole in Blood and BAL-fluid After Inhalation and Oral Administration
NCT03066011	Not specified	COMPLETED	Registry of Patients Treated With Systemic Mold-Active Triazoles
NCT03138785	Not specified	UNKNOWN	UVX as an Adjuvant in the Treatment of Fungal Keratitis

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

PharmGKB dosing guidelines (3) — CPIC / DPWG genotype-guided dosing for this drug (drug × pharmacogene):

Guideline	Source	Gene(s)	Dosing	Recommendation
Annotation of DPWG Guideline for voriconazole and CYP2C19	DPWG	CYP2C19	yes	yes
Annotation of CPIC Guideline for voriconazole and CYP2C19	CPIC	CYP2C19	yes	yes
Annotation of AusNZ Guideline for voriconazole and CYP2C19	AusNZ	CYP2C19	yes	yes

PharmGKB also curates 4 clinical and 169 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).