Ganciclovir

Summary

Ganciclovir (CHEMBL182) is an approved small-molecule antiviral drug (ATC S01AD09); indicated across 20 conditions including viral infectious disease and cytomegalovirus retinitis.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: S01AD09 (+1 more)
• Indications: 20 conditions
• Clinical trials: 94
• Chemistry: 255.23 Da · C9H13N5O4

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL182
Name	Ganciclovir
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	135398740
ChEBI	CHEBI:465284
ATC	S01AD09, J05AB06
Molecular formula	C9H13N5O4
Molecular weight	255.23
InChIKey	IRSCQMHQWWYFCW-UHFFFAOYSA-N

SMILES: C1=NC2=C(N1COC(CO)CO)N=C(NC2=O)N

IUPAC name: 2-amino-9-(1,3-dihydroxypropan-2-yloxymethyl)-1H-purin-6-one

ChEBI definition: An oxopurine that is guanine substituted by a [(1,3-dihydroxypropan-2-yl)oxy]methyl group at position 9. Ganciclovir is an antiviral drug used to treat or prevent AIDS-related cytomegalovirus infections.

Pharmacological roles (ChEBI): antiviral drug, antiinfective agent.

Also known as: BW 759U, BW-759U, Cymevene, Cytovene, Ganciclovir, Gancyclovir, Ganzyk-rtu, NSC-759656, RS-21592, Valganciclovir hydrochloride impurity a, Virgan, Vitrasert

Parent form; salt/anhydrous children: CHEMBL1200850

Patent coverage: 37,528 distinct patent families (138,136 SureChEMBL compound mentions), from 3 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

Broader ChEMBL bioactivity targets: 11 (assay-derived). Sample: Transitional endoplasmic reticulum ATPase, Purine nucleoside phosphorylase, Survival motor neuron protein, Prelamin-A/C, RecQ-like DNA helicase BLM, Endonuclease 4, Menin/Histone-lysine N-methyltransferase MLL, Nuclear factor NF-kappa-B p105 subunit, Cytochrome P450 3A4, Aldehyde dehydrogenase 1A1.

Bioactivity

ChEMBL activities: 11 potent at pChembl ≥ 5 of 18 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
BLM	8.49	Potency	3.2	nM	CHEMBL_ACT_4745946
BLM	8.49	Potency	3.2	nM	CHEMBL_ACT_4925738
VCP	5.89	IC50	1300	nM	CHEMBL_ACT_24663324
SMN1	5.8	Potency	1585	nM	CHEMBL_ACT_3894354
LMNA	5.5	Potency	3162	nM	CHEMBL_ACT_3649752
HIF1A	5.4	Potency	3981	nM	CHEMBL_ACT_4128797
HIF1A	5.4	Potency	3981	nM	CHEMBL_ACT_4518590
MEN1	5	Potency	10000	nM	CHEMBL_ACT_3620286
NFKB1	5	Potency	10000	nM	CHEMBL_ACT_3675704
MEN1	5	Potency	10000	nM	CHEMBL_ACT_4552572
NFKB1	5	Potency	10000	nM	CHEMBL_ACT_4588886

Target pathways

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

Indications & clinical

Indications

20 indications (4 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
viral infectious disease	4	MONDO:0005108	EFO:0000763
cytomegalovirus retinitis	4	MONDO:0000878	EFO:1001302
eye infectious disorder	4	MONDO:0043885	EFO:1001888
HIV infectious disease	4	MONDO:0005109	EFO:0000764
cytomegalovirus infection	3	MONDO:0005132	EFO:0001062
conjunctivitis	3	MONDO:0003799	EFO:0009450
hepatocellular carcinoma	2	MONDO:0007256	EFO:0000182
glioblastoma	2	MONDO:0018177	EFO:0000519
lymphoma	2	MONDO:0005062	EFO:0000574
peritoneal neoplasm	2	MONDO:0006901	MONDO:0002087
fallopian tube neoplasm	2	MONDO:0021092	MONDO:0002158
ovarian cancer	2	MONDO:0008170	MONDO:0008170
graft versus host disease	2	MONDO:0013730	MONDO:0013730
type 1 diabetes mellitus	2	MONDO:0005147	MONDO:0005147
cutaneous melanoma	1	MONDO:0005012	EFO:0000389
mesothelioma	1	MONDO:0005065	EFO:0000588
neuroblastoma	1	MONDO:0005072	EFO:0000621
kidney disorder	1	MONDO:0005240	EFO:0003086
central nervous system neoplasm	1	MONDO:0006130	EFO:1000158

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

Clinical trials

Total trials: 94.

Phase distribution

Phase	Trials
Not specified	31
PHASE3	18
PHASE2	16
PHASE1	16
PHASE4	10
PHASE1/PHASE2	3

Top trials by phase / activity

NCT	Phase	Status	Title
NCT07235683	PHASE4	RECRUITING	Combination Letermovir and Standard of Care Antiviral for Enhanced Antiviral Response in Cytomegalovirus Infection in Lung Transplant Recipients
NCT07450365	PHASE4	NOT_YET_RECRUITING	Evaluation of Long-term Efficacy of 4 to 6-month Course Antiviral Therapy for Neurodevelopmental Impairments Caused by Congenital Cytomegalovirus Infection
NCT00000894	PHASE4	COMPLETED	Comparison of Two Drugs, Cidofovir and Ganciclovir, in Treating Patients With AIDS Who Have CMV Retinitis
NCT00264368	PHASE4	TERMINATED	Ganciclovir Pharmacokinetics in Patients Undergoing Continuous Renal Replacement Therapy
NCT00373165	PHASE4	COMPLETED	Prophylaxis With Ganciclovir Improves Graft Survival in Renal Allograft Recipients
NCT00431353	PHASE4	COMPLETED	VICTOR Study - A Study of Valcyte (Valganciclovir po) Compared to Ganciclovir iv in Patients With Cytomegalovirus (CMV) Disease Who Are Solid Organ Transplant Recipients
NCT01446445	PHASE4	COMPLETED	Individualization of Ganciclovir and Valganciclovir Doses Using Bayesian Prediction in Renal Transplant Patients.
NCT01533480	PHASE4	COMPLETED	A Placebo Controlled Comparison of Topical Zirgan Versus Genteal Gel for the Treatment of Adenovirus Conjunctivitis
NCT02152358	PHASE4	COMPLETED	PTH - Preemptive Treatment for Herpesviridae
NCT03004261	PHASE4	COMPLETED	CMV-CTL for the Treatment of CMV Infection After HSCT
NCT00000134	PHASE3	COMPLETED	Studies of the Ocular Complications of AIDS (SOCA)–Cytomegalovirus Retinitis Retreatment Trial (CRRT)
NCT00000136	PHASE3	COMPLETED	Studies of the Ocular Complications of AIDS (SOCA)–Foscarnet-Ganciclovir CMV Retinitis Trial (FGCRT)
NCT00000143	PHASE3	COMPLETED	Studies of Ocular Complications of AIDS (SOCA)–Ganciclovir-Cidofovir CMV Retinitis Trial (GCCRT)
NCT00000688	PHASE3	COMPLETED	A Randomized, Controlled Study of Intravenous Ganciclovir Therapy for Peripheral Cytomegalovirus Retinitis in Patients With AIDS
NCT00000698	PHASE3	COMPLETED	A Treatment Protocol for the Use of Intravenous Ganciclovir in AIDS Patients With Immediately Sight-Threatening CMV Retinitis
NCT00001100	PHASE3	COMPLETED	A Phase III Study to Evaluate the Safety and Efficacy of Ganciclovir (Dihydroxypropoxymethyl Guanine [DHPG]) Treatment of Symptomatic Central Nervous System (CNS) Congenital Cytomegalovirus (CMV) Infections.
NCT00002070	PHASE3	COMPLETED	Phase III Ganciclovir +/- rGM-CSF for AIDS-Related CMV Retinitis
NCT00002377	PHASE3	COMPLETED	A Comparison of Valganciclovir and Ganciclovir in the Treatment of Cytomegalovirus (CMV) of the Eyes
NCT00004278	PHASE3	COMPLETED	Phase III Randomized, Controlled Study of Ganciclovir for Symptomatic Congenital Cytomegalovirus Infection
NCT00016068	PHASE3	COMPLETED	Valganciclovir to Prevent Cytomegalovirus Infection in Patients Following Donor Stem Cell Transplantation
NCT00241345	PHASE3	TERMINATED	Trial of Preemptive Treatment With Oral Valganciclovir Compared With Intravenous (IV) Ganciclovir for Cytomegalovirus Infection After Bone Marrow or Peripheral Blood Stem Cell Transplant
NCT00372229	PHASE3	COMPLETED	A Study of Valcyte (Valganciclovir) CMV Prophylaxis After Renal Transplantation
NCT00497796	PHASE3	COMPLETED	Maribavir Versus Oral Ganciclovir For The Prevention of Cytomegalovirus (CMV) Disease in Liver Transplant Recipients
NCT01185223	PHASE3	TERMINATED	Study Comparing Valganciclovir Versus Ganciclovir in Patients Following Allogeneic Stem Cell Transplantation
NCT01600365	PHASE3	UNKNOWN	Evaluate the Use of Ganciclovir Gel 0.3% for the Treatment of Conjunctivitis Caused by Adenovirus
NCT02931539	PHASE3	COMPLETED	Efficacy and Safety Study of Maribavir Treatment Compared to Investigator-assigned Treatment in Transplant Recipients With Cytomegalovirus (CMV) Infections That Are Refractory or Resistant to Treatment With Ganciclovir, Valganciclovir, Foscarnet, or Cidofovir
NCT03699254	PHASE3	COMPLETED	Clinical Trial of Efficacy and Safety of the Combination of Reduced Duration Prophylaxis Followed by Immuno-guided Prophylaxis in Lung Transplant Recipients.
NCT06341686	PHASE3	UNKNOWN	Evaluation of the Prophylactic Use of Letermovir in Kidney Transplant Recipients at Risk of Cytomegalovirus Infection
NCT06853184	PHASE2	RECRUITING	Study Evaluating the Efficacy and Safety of Artesunate
NCT06971913	PHASE2	NOT_YET_RECRUITING	Efficacy and Safety of Low-dose IL-2 in SLE Patients With CMV Viremia
NCT07513623	PHASE2	NOT_YET_RECRUITING	Systemic and Topical Antiviral Control of Cytomegalovirus Anterior Uveitis: Treatment Outcomes - Trials I and II
NCT00000766	PHASE2	COMPLETED	CMV Retinitis Retreatment Trial
NCT00001034	PHASE2	COMPLETED	The Safety and Effectiveness of Ganciclovir in the Prevention of Cytomegalovirus (CMV) of the Eyes and Disease of the Stomach and Intestines in Patients With HIV
NCT00001061	PHASE2	COMPLETED	Comparison of Two Methods in the Treatment of Cytomegalovirus of the Eyes in Patients With AIDS
NCT00002156	PHASE2	COMPLETED	A Randomized Comparison of Intravitreal ISIS 2922 Plus Ganciclovir Versus Ganciclovir as Treatment for Patients With Cytomegalovirus Retinitis ( CMVR )
NCT00005025	PHASE2	UNKNOWN	Gene Therapy in Treating Women With Refractory or Relapsed Ovarian Epithelial Cancer, Fallopian Tube Cancer, or Peritoneal Cancer
NCT00006264	PHASE2	COMPLETED	Zidovudine Plus Interleukin-2 and Ganciclovir in Treating Patients With AIDS-Related Primary Central Nervous System Lymphoma
NCT00014911	PHASE2	COMPLETED	Islet Transplantation for Type 1 Diabetes
NCT00031434	PHASE1/PHASE2	COMPLETED	Valganciclovir in Congenital CMV Infants
NCT00141037	PHASE1/PHASE2	COMPLETED	Steroid-Free Versus Steroid-Based Immunosuppression in Pediatric Renal (Kidney) Transplantation

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

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