Zoledronic Acid Anhydrous

Summary

Zoledronic Acid Anhydrous (CHEMBL924) is an approved small-molecule bone density conservation agent targeting FDPS; indicated across 8 conditions including plasma cell myeloma and bone disorder.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• Targets: 1 (FDPS)
• Indications: 8 conditions
• Clinical trials: 294
• Chemistry: 272.09 Da · C5H10N2O7P2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL924
Name	Zoledronic Acid Anhydrous
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	68740
ChEBI	CHEBI:46557
Molecular formula	C5H10N2O7P2
Molecular weight	272.09
InChIKey	XRASPMIURGNCCH-UHFFFAOYSA-N

SMILES: C1=CN(C=N1)CC(O)(P(=O)(O)O)P(=O)(O)O

IUPAC name: (1-hydroxy-2-imidazol-1-yl-1-phosphonoethyl)phosphonic acid

ChEBI definition: An imidazole compound having a 2,2-bis(phosphono)-2-hydroxyethane-1-yl substituent at the 1-position.

Pharmacological roles (ChEBI): bone density conservation agent.

Also known as: Anhydrous zoledronic acid, Zoledronate, Zoledronic acid anhydrous, zoledronate, ZOLEDRONIC ACID ANHYDROUS, Zoledronic acid

Parent form; salt/anhydrous children: CHEMBL2103912, CHEMBL3989655, CHEMBL4303669, CHEMBL4746555

Patent coverage: 10,532 distinct patent families (42,100 SureChEMBL compound mentions), from 1 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).

Gene	Target	Action	pAffinity	Cancer dependency	UniProt
FDPS	farnesyl diphosphate synthase	Inhibition	10.15	72.4%	P14324

Broader ChEMBL bioactivity targets: 1 (assay-derived). Sample: Farnesyl pyrophosphate synthase.

Bioactivity

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

Target	pChembl	Type	Value	Unit	Activity ID
FDPS	6.26	IC50	550	nM	CHEMBL_ACT_26120846

Target pathways

Aggregated over 1 target gene(s): FDPS.

Top Reactome pathways

3 total, by targets touching each:

Pathway	Targets	Genes
Cholesterol biosynthesis	1	FDPS
Activation of gene expression by SREBF (SREBP)	1	FDPS
Lanosterol biosynthesis	1	FDPS

Dominant GO biological processes

GO term	Targets
cholesterol biosynthetic process	1
geranyl diphosphate biosynthetic process	1
trans, trans-farnesyl diphosphate biosynthetic process	1
lipid metabolic process	1
steroid biosynthetic process	1
steroid metabolic process	1
cholesterol metabolic process	1
isoprenoid biosynthetic process	1
sterol biosynthetic process	1

Indications & clinical

Indications

7 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
plasma cell myeloma	3	MONDO:0009693	EFO:0001378
bone disorder	3	MONDO:0005381	EFO:0004260
complex regional pain syndrome type 1	3	MONDO:0011441	EFO:1001147
complex regional pain syndrome	3	MONDO:0019369	EFO:1001998
renal cell carcinoma	2	MONDO:0005086	EFO:0000681
kidney cancer	2	MONDO:0002367	MONDO:0002367
breast neoplasm	2	MONDO:0021100	MONDO:0007254

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

Clinical trials

Total trials: 294.

Phase distribution

Phase	Trials
PHASE3	90
PHASE4	74
PHASE2	58
Not specified	32
PHASE1	20
PHASE2/PHASE3	11
PHASE1/PHASE2	9

Top trials by phase / activity

NCT	Phase	Status	Title
NCT03924414	PHASE4	ACTIVE_NOT_RECRUITING	Trial of Parkinson’s And Zoledronic Acid
NCT05058976	PHASE4	ACTIVE_NOT_RECRUITING	Romosozumab Use to Build Skeletal Integrity
NCT05655013	PHASE4	ACTIVE_NOT_RECRUITING	Treatment With Zoledronate Subsequent to Denosumab in Osteoporosis 2 (ZOLARMAB2)
NCT05666310	PHASE4	ACTIVE_NOT_RECRUITING	Muscle Impact of Treating Osteoporosis
NCT06059222	PHASE4	RECRUITING	The Optimised Use of Romozosumab Study
NCT06118905	PHASE4	RECRUITING	Preserving Geriatric Muscle With an Osteoporosis Medication
NCT06762730	PHASE4	RECRUITING	Zoledronic Acid for the Prevention of TDF-sparing ART-induced Bone Mass Density Loss in Treatment-naive HIV Positive Individuals
NCT06859580	PHASE4	RECRUITING	Bisphosphonate Prior to Parathyroidectomy in Primary Hyperparathyroidism
NCT07281586	PHASE4	RECRUITING	Step-down Therapy After Long-term Osteoporosis Treatment
NCT00029224	PHASE4	COMPLETED	Treatment With Zoledronic Acid in Patients With Breast Cancer, Multiple Myeloma, and Prostate Cancer With Cancer Related Bone Lesions
NCT00063609	PHASE4	COMPLETED	The Effect of Zoledronic Acid on Bone Loss in Prostate Cancer Patients Undergoing Androgen Deprivation Therapy
NCT00099541	PHASE4	COMPLETED	Non-small Cell Lung Cancer Registry
NCT00104104	PHASE4	COMPLETED	A Multiple Myeloma Trial in Patients With Bone Metastases
NCT00114556	PHASE4	COMPLETED	The Effect of Zoledronic Acid on Bone Density in Liver Transplant Patients
NCT00172003	PHASE4	COMPLETED	Effect of Zoledronic Acid in Patients With Renal Cell Cancer and Bone Metastasis
NCT00172016	PHASE4	COMPLETED	A Study to Evaluate the Efficacy and Tolerability of Zoledronic Acid in Patients With Metastatic Prostate Cancer Who Can be Treated With a Group of Medications Known as Bisphosphonates
NCT00172029	PHASE4	COMPLETED	Study Comparing Full-dose Radiotherapy Versus Reduced Dose in the Management of Bone Metastasis in Patients With Breast Cancer Receiving Zoledronic Acid
NCT00219219	PHASE4	COMPLETED	Zoledronic Acid in the Prevention of Skeletal-related Events in Hormone Refractory and Hormone-sensitive Prostate Cancer Patients With Bone Metastases
NCT00219271	PHASE4	COMPLETED	Effect Of Zoledronic Acid On Circulating And Bone Marrow-Residing Prostate Cancer Cells In Patients With Clinically Localized Prostate Cancer
NCT00237146	PHASE4	COMPLETED	Study to Evaluate Zoledronic Acid on Quality of Life and Skeletal-related Events as Adjuvant Treatment in Patients With Hormone-naïve Prostate Cancer and Bone Metastasis Who Have Undergone Orchiectomy
NCT00237159	PHASE4	COMPLETED	Effect of Zoledronic Acid in Patients With Prostate Cancer and Bone Metastasis
NCT00241111	PHASE4	COMPLETED	Assessment of the Efficacy, Tolerability and Pharmaco-economic Impact of Zoledronic Acid Treatment in Prostate Cancer With Bone Metastasis
NCT00242528	PHASE4	WITHDRAWN	Open-label Study, to Evaluate the Safety and Tolerability of Zoledronic Acid in Patients With Bone Lesions Secondary to Multiple Myeloma.
NCT00242554	PHASE4	COMPLETED	Open-label Phase IV Clinical Trial to Evaluate the Safety and Tolerability of Zoledronic Acid in Patients With Prostate Cancer and Bone Metastases
NCT00333229	PHASE4	TERMINATED	A Study of Zoledronic Acid in the Prevention of Cancer Therapy-induced Bone Loss
NCT00334139	PHASE4	COMPLETED	Effect of Zoledronic Acid on Bone Metabolism in Patients With Bone Metastasis and Prostate or Breast Cancer
NCT00346242	PHASE4	COMPLETED	Evaluation of Efficacy of Zoledronic Acid in Patients With Haemoglobin Syndromes (Thalassemia and Sicle Cell Anaemia) and Risk of Skeletal Events
NCT00391690	PHASE4	COMPLETED	Evaluation of Bone Markers as Diagnostic Tools for Early Detection of Bone Metastases in Patients With High Risk Prostate Cancer
NCT00431444	PHASE4	COMPLETED	Effects of Zoledronic Acid and Raloxifene on Bone Turnover Markers in Postmenopausal Women With Low Bone Mineral Density
NCT00434317	PHASE4	COMPLETED	Assessment of Pain and Quality of Life in Breast and Prostate Cancer Patients With Bone Metastases
NCT00434447	PHASE4	COMPLETED	Long Term Efficacy and Safety of Zoledronic Acid Treatment in Patients With Bone Metastases
NCT00622505	PHASE4	COMPLETED	Zoledronic Acid Treatment (Every 4 or 12 Weeks) to Prevent Skeletal Complications in Advanced Multiple Myeloma Participants
NCT00740129	PHASE4	COMPLETED	Re-treatment of Participants With Paget’s Disease Using Zoledronic Acid
NCT00745485	PHASE4	COMPLETED	Feasibility of Doctors’ Rooms-based Infusion of Zoledronic Acid
NCT00774020	PHASE4	COMPLETED	Efficacy and Safety of Single Dose of 5 mg Zoledronic Acid in Chinese Patients With Paget’s Disease of Bone (PDB)
NCT00795483	PHASE4	COMPLETED	Efficacy And Security Of Annual And Biennial Zoledronic Acid For Osteoporosis Treatment In An HIV-Infected Patients’ Cohort
NCT00912938	PHASE4	UNKNOWN	Evaluate the Relationship of Bone Remodeling Markers for Skeletal Complications in Metastatic Breast Cancer Patients
NCT00927186	PHASE4	COMPLETED	Effects of Teriparatide or Zoledronic Acid Treatment on Bone in Postmenopausal Osteoporotic Women
NCT01087008	PHASE4	COMPLETED	Zoledronic Acid in Patients With Multiple Myeloma and Asymptomatic Biochemical Relapse
NCT01129336	PHASE4	COMPLETED	Effect of Zoledronic Acid as Anti-Cancer Treatment in Metastatic Breast Cancer Patients
NCT01153425	PHASE4	COMPLETED	µMRI of Therapeutic Intervention in Postmenopausal Osteoporosis
NCT01218035	PHASE4	COMPLETED	Efficacy of Zoledronic Acid in Enhancement of Early Stability of Cementless Primary Hip Prosthesis
NCT01267279	PHASE4	COMPLETED	Effect of Zoledronic Acid on Femoral Bone Loss Following Total Hip Arthroplasty
NCT01310465	PHASE4	UNKNOWN	The Effect of Zoledronic Acid to Bone Fusion and Bone Metabolism of Patients With Lumbar Degenerative Disease After Lumbar Interbody Fusion
NCT01675089	PHASE4	COMPLETED	Zoledronic Acid to Prevent Bone Loss After Kidney Transplantation
NCT01702415	PHASE4	WITHDRAWN	Zoledronic Acid in Cystic Fibrosis
NCT01732770	PHASE4	COMPLETED	Safety and Efficacy Study to Evaluate Denosumab Compared With Zoledronic Acid in Postmenopausal Women With Osteoporosis
NCT01907880	PHASE4	COMPLETED	The ODYSSEY TRIAL Phase IV Trial Evaluating the Palliative Benefit of Pamidronate or Zoledronic Acid in Breast Cancer
NCT02042872	PHASE4	COMPLETED	Zoledronic Acid Administration in Acute Spinal Cord Injury
NCT02176382	PHASE4	COMPLETED	Denosumab and Teriparatide Study (DATA-HD and DATA-EX)
NCT02286830	PHASE4	COMPLETED	Prolonged Protection From Bone Disease in Multiple Myeloma
NCT02333344	PHASE4	WITHDRAWN	The Efficacy Study of Aclasta on Prosthetic Fixation in Postmenopausal Women After THA
NCT02480634	PHASE4	UNKNOWN	Zoledronic Acid Combined Radiotherapy for Bone Metastasis of Non-small Cell Lung Cancer
NCT02499237	PHASE4	COMPLETED	Zoledronic Acid to Maintain Bone Mass After Denosumab Discontinuation
NCT02589600	PHASE4	COMPLETED	Zoledronic Acid for Osteoporotic Fracture Prevention (ZEST II)
NCT02721433	PHASE4	COMPLETED	4-weekly Versus 12-weekly Administration of Bone-targeted Agents in Patients With Bone Metastases
NCT02808988	PHASE4	COMPLETED	Changes of GCF Levels of RANKL and Osteoprotegerin in Postmenopausal Osteoporosis
NCT03087851	PHASE4	COMPLETED	Treatment With Zoledronic Acid Subsequent to Denosumab in Osteoporosis
NCT03424239	PHASE4	COMPLETED	Pilot Study for Zoledronic Acid to Prevent Bone Loss After Bariatric Surgery
NCT03664687	PHASE4	COMPLETED	Comparing a Single-Dose vs. Twice Yearly Zoledronate in Patients With Early Stage Breast Cancer (REaCT-ZOL)
NCT03735537	PHASE4	COMPLETED	Treatment of Osteogenesis Imperfecta With Parathyroid Hormone and Zoledronic Acid
NCT03868033	PHASE4	COMPLETED	Denosumab Sequential Therapy
NCT03932071	PHASE4	UNKNOWN	Zoledronic Acid in Decrease the Lung Metastatic Rate of Osteosarcoma
NCT04085419	PHASE4	UNKNOWN	Osteoporosis in Primary Hyperparathyroidism
NCT04087096	PHASE4	COMPLETED	Denosumab to Prevent High-Turnover Bone Loss After Bariatric Surgery
NCT04115397	PHASE4	UNKNOWN	Bisphosphonates for the Treatment of Seropositive Musculoskeletal Complaints
NCT04597931	PHASE4	UNKNOWN	Evaluation of Romosozumab vs. Zoledronic Acid Effect in Patients With Spinal Cord Injury and Low Bone Mineral Density
NCT05025293	PHASE4	UNKNOWN	Zoledronate Early to Hip Fracture Patients - Safe and Effective?
NCT05091099	PHASE4	COMPLETED	The Optimal Sequential Therapy After Long Term Denosumab Treatment
NCT05361408	PHASE4	COMPLETED	A Clinical Trial to Evaluate Efficacy of Once or Twice ZOledronic Acid After Different Duration of denOsumMab Administration in Postmenopausal Women With Osteoporosis (ZOOM Study)
NCT05598606	PHASE4	COMPLETED	Denosumab vs Zoledronate After Vertebroplasty
NCT05743179	PHASE4	UNKNOWN	The Effect of Zoledronate on the Prevention of Pneumonia in Hip Fracture Patients
NCT06051344	PHASE4	UNKNOWN	Effect of Zoledronic Acid in Primary Knee Osteoarthritis
NCT06135298	PHASE4	COMPLETED	FARE Augmentation of Proximal Femoral Fractures With CaS/HA and Systemic ZA
NCT00268476	PHASE2/PHASE3	ACTIVE_NOT_RECRUITING	Systemic Therapy in Advancing or Metastatic Prostate Cancer: Evaluation of Drug Efficacy
NCT00470223	PHASE3	ACTIVE_NOT_RECRUITING	Combined Chemotherapy With or Without Zoledronic Acid for Patients With Osteosarcoma
NCT03295981	PHASE3	RECRUITING	Local Bisphosphonate Effect on Recurrence Rate in Extremity Giant Cell Tumor of Bone
NCT06221072	PHASE3	NOT_YET_RECRUITING	A Study to Assess the Prevention of Bone-related Events in Patients With Bone Metastases From Malignant Solid Tumors Treated With JMT103 Compared to Zoledronic Acid
NCT06533865	PHASE3	RECRUITING	Romosozumab as an Adjunct to Physiologic Estrogen Replacement in Functional Hypothalamic Amenorrhea
NCT00003884	PHASE3	COMPLETED	Zoledronate in Treating Patients With Solid Tumors That Have Spread to the Bone
NCT00005073	PHASE3	TERMINATED	Zoledronate Plus Standard Therapy Compared With Placebo Plus Standard Therapy to Prevent Bone Metastases in Patients With Recurrent Prostate Cancer That Has No Symptoms
NCT00022087	PHASE3	COMPLETED	Zoledronate, Calcium, and Vitamin D in Preventing Bone Loss in Women Receiving Adjuvant Chemotherapy for Breast Cancer
NCT00033332	PHASE3	COMPLETED	Dexamethasone With or Without Thalidomide in Treating Patients With Newly Diagnosed Multiple Myeloma
NCT00046254	PHASE3	COMPLETED	Evaluating the Ability of Zoledronic Acid to Reduce the Rate of Subsequent Osteoporotic Fractures After a Hip Fracture
NCT00049452	PHASE3	COMPLETED	Zoledronate in Preventing Bone Loss in Premenopausal Women Receiving Chemotherapy After Surgery For Early Stage Breast Cancer
NCT00049829	PHASE3	COMPLETED	HORIZON-PFT: Pivotal Fracture Trial
NCT00050011	PHASE3	COMPLETED	Zoledronic Acid - Letrozole Adjuvant Synergy Trial (ZFAST) - Cancer Treatment Related Bone Loss in Postmenopausal Women With Estrogen Receptor Positive and/or Progesterone Receptor Positive Breast Cancer Receiving Adjuvant Hormonal Therapy
NCT00051636	PHASE3	COMPLETED	Safety and Efficacy Trial With Zoledronic Acid for the Treatment of Paget’s Disease of Bone, Including an Extended Observation Period
NCT00058188	PHASE3	TERMINATED	Zolendronate for the Prevention of Bone Loss in Men w/ Prostate CA on Long-Term Androgen Deprivation
NCT00072020	PHASE3	UNKNOWN	Chemotherapy and/or Hormone Therapy With or Without Zoledronate in Treating Women With Stage II or Stage III Breast Cancer
NCT00079001	PHASE3	COMPLETED	Zoledronate in Preventing Skeletal (Bone)-Related Events in Men Who Are Receiving Androgen Deprivation Therapy For Prostate Cancer and Bone Metastases
NCT00086268	PHASE3	COMPLETED	Non-small Cell Lung Cancer Study US75 (Z-PACT)
NCT00097812	PHASE3	COMPLETED	Switching Osteoporosis Patients Currently on Oral Bisphosphonate to Zoledronic Acid
NCT00097825	PHASE3	COMPLETED	Efficacy and Safety of Zoledronic Acid for the Treatment of Osteoporosis in Men
NCT00099177	PHASE3	TERMINATED	A Study to Assess the Efficacy of Intravenous/Oral Bondronat (Ibandronate) in Patients With Metastatic Bone Disease Experiencing Moderate to Severe Pain
NCT00099203	PHASE3	TERMINATED	A Study to Assess the Efficacy of Intravenous Bondronat (Ibandronate) in Patients With Malignant and Painful Bone Disease
NCT00100555	PHASE3	COMPLETED	A Study of the Clinical Safety, Tolerability, and Efficacy of Zoledronic Acid Compared to an Oral Bisphosphonate
NCT00100620	PHASE3	COMPLETED	Efficacy and Safety of Zoledronic Acid in the Prevention and Treatment of Corticosteroid Induced Osteoporosis
NCT00103740	PHASE3	COMPLETED	Safety and Efficacy Trial With Zoledronic Acid for the Treatment of Paget’s Disease of Bone, Including an Extended Observation Period
NCT00107263	PHASE3	COMPLETED	Zoledronate in Preventing Bone Loss in Postmenopausal Women Who Are Receiving Letrozole for Stage I, Stage II, or Stage IIIA Breast Cancer

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 4 clinical and 13 variant annotation(s) for this drug (gene-keyed; see PharmGKB).

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
ALENDRONIC ACID	ChEMBL + PubChem	Phase 4 (approved)	FDPS
IBANDRONIC ACID	ChEMBL + PubChem	Phase 4 (approved)	FDPS
PAMIDRONIC ACID	ChEMBL + PubChem	Phase 4 (approved)	FDPS
RISEDRONIC ACID	ChEMBL + PubChem	Phase 4 (approved)	FDPS
ALENDRONATE	ChEMBL	Phase 4 (approved)	FDPS
MINODRONIC ACID	ChEMBL	Phase 4 (approved)	FDPS
NERIDRONIC ACID	ChEMBL	Phase 3	FDPS
PYROPHOSPHORIC ACID	ChEMBL + PubChem	Phase 2 (approved)	FDPS
INCADRONIC ACID	ChEMBL	Phase 2	FDPS
PIRIDRONIC ACID	ChEMBL	Phase 2	FDPS
Lovastatin	PubChem	Approved	FDPS