Pamidronic Acid

Summary

Pamidronic Acid (CHEMBL834) is an approved small molecule (ATC M05BA03) targeting FDPS; indicated across 5 conditions including bone disorder and plasma cell myeloma.

At a glance

• Status: Approved (max clinical phase 4)
• Modality: Small molecule
• ATC class: M05BA03
• Targets: 1 (FDPS)
• Indications: 5 conditions
• Clinical trials: 30
• Chemistry: 235.07 Da · C3H11NO7P2

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL834
Name	Pamidronic Acid
Type	Small molecule
Max phase	4
FDA approved	yes
PubChem CID	4674
ATC	M05BA03
Molecular formula	C3H11NO7P2
Molecular weight	235.07
InChIKey	WRUUGTRCQOWXEG-UHFFFAOYSA-N

SMILES: C(CN)C(O)(P(=O)(O)O)P(=O)(O)O

IUPAC name: (3-amino-1-hydroxy-1-phosphonopropyl)phosphonic acid

Also known as: Acide pamidronique, Acido pamidronico, Pamidronate, Pamidronic acid, Ribodroat, pamidronate, SID29215372, SID29217620, ACIDE_PAMIDRONIQUE, SID170464689, SID144205217, PAMIDRONIC ACID

Parent form; salt/anhydrous children: CHEMBL676, CHEMBL3989401

Patent coverage: 12,727 distinct patent families (52,185 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	6.7	72.4%	P14324

Broader ChEMBL bioactivity targets: 7 (assay-derived). Sample: Prelamin-A/C, Ferritin light chain, Farnesyl pyrophosphate synthase, cGMP-inhibited 3’,5’-cyclic phosphodiesterase 3A, Beta-3 adrenergic receptor, 3’,5’-cyclic-AMP phosphodiesterase 4D, Hypoxanthine-guanine phosphoribosyltransferase.

Bioactivity

ChEMBL activities: 11 potent at pChembl ≥ 5 of 16 total. Top 30 by potency (10 = 0.1 nM, 6 = 1 µM):

Target	pChembl	Type	Value	Unit	Activity ID
FDPS	7.25	Ki	55.9	nM	CHEMBL_ACT_2155003
FDPS	6.75	Ki	180	nM	CHEMBL_ACT_1089168
FDPS	6.7	IC50	200	nM	CHEMBL_ACT_12049463
FDPS	6.7	IC50	200	nM	CHEMBL_ACT_900263
FDPS	6.48	Ki	331.4	nM	CHEMBL_ACT_2155002
FDPS	6.45	IC50	353.2	nM	CHEMBL_ACT_2155001
FDPS	6.3	IC50	500	nM	CHEMBL_ACT_24973370
FDPS	5.73	IC50	1862	nM	CHEMBL_ACT_1089169
FDPS	5.72	IC50	1900	nM	CHEMBL_ACT_1089167
PDE3A	5.72	AC50	1894	nM	CHEMBL_ACT_25191037
FDPS	5.71	IC50	1932	nM	CHEMBL_ACT_2154997

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

5 indications (1 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
bone disorder	4	MONDO:0005381	EFO:0004260
plasma cell myeloma	3	MONDO:0009693	EFO:0001378
breast neoplasm	2	MONDO:0021100	MONDO:0007254
osteoporosis	1	MONDO:0005298	EFO:0003882

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

Clinical trials

Total trials: 30.

Phase distribution

Phase	Trials
PHASE4	9
PHASE2	6
Not specified	5
PHASE1	4
PHASE3	3
PHASE1/PHASE2	3

Top trials by phase / activity

NCT	Phase	Status	Title
NCT00108394	PHASE4	COMPLETED	Osteopenia and Renal Osteodystrophy: Evaluation and Management
NCT00128297	PHASE4	COMPLETED	Pamidronate Administration in Breast Cancer Patients With Bone Metastases
NCT00159419	PHASE4	COMPLETED	Bisphosphonate Therapy for Osteogenesis Imperfecta
NCT00738257	PHASE4	COMPLETED	A Prospective, Multicentre, Open-label Randomised Controlled Study to Investigate the Effectiveness of Pamidronate in the Prevention of Bone Loss in de Novo Renal Transplant Patients.
NCT01718951	PHASE4	COMPLETED	Golimumab Versus Pamidronate for the Treatment of Axial Spondyloarthropathy: a 48-week Trial
NCT01907880	PHASE4	COMPLETED	The ODYSSEY TRIAL Phase IV Trial Evaluating the Palliative Benefit of Pamidronate or Zoledronic Acid in Breast Cancer
NCT02101164	PHASE4	WITHDRAWN	Evaluate Time Associated With the Preparation & Administration of Denosumab/Pamidronate in Patients With Solid Tumors and Metastatic Bone Disease
NCT02313727	PHASE4	UNKNOWN	Combined Treatment With TNF Inhibitor and Pamidronate in AS Patients: Effect on the Radiographic Progression
NCT02721433	PHASE4	COMPLETED	4-weekly Versus 12-weekly Administration of Bone-targeted Agents in Patients With Bone Metastases
NCT00005901	PHASE3	COMPLETED	Pamidronate to Treat Osteogenesis Imperfecta in Children
NCT00033332	PHASE3	COMPLETED	Dexamethasone With or Without Thalidomide in Treating Patients With Newly Diagnosed Multiple Myeloma
NCT00376883	PHASE3	COMPLETED	Pamidronate Prophylaxis in Multiple Myeloma 30 mg/Month Versus 90 mg/Month
NCT00004088	PHASE2	COMPLETED	Combination Chemo, Peripheral Stem Cell Transplant, Biological Therapy, Pamidronate and Thalidomide for Multiple Myeloma
NCT00004906	PHASE2	COMPLETED	Combination Chemotherapy and Peripheral Stem Cell Transplantation in Treating Women With Metastatic Breast Cancer
NCT00022841	PHASE1/PHASE2	TERMINATED	Prevention of Steroid-Induced Osteoporosis in Children
NCT00083382	PHASE2	COMPLETED	Combination Bisphosphonate and Anti-Angiogenesis Therapy With Pamidronate and Thalidomide
NCT00083408	PHASE2	COMPLETED	Combination Bisphosphonate and Anti-Angiogenesis Therapy With Pamidronate and Thalidomide
NCT00482378	PHASE1/PHASE2	COMPLETED	Samarium Sm 153 Lexidronam Pentasodium Combined With Zoledronic Acid or Pamidronate in Treating Patients With Relapsed or Refractory Multiple Myeloma and Bone Pain
NCT01210599	PHASE1/PHASE2	COMPLETED	A Pilot Trial of IV Pamidronate for Low Back Pain
NCT01799616	PHASE2	UNKNOWN	Efficiency and Safety Study of Pamidronate in Inflammatory Back Pain Due to Degenerative Disk Disease
NCT02074631	PHASE2	COMPLETED	Prevention of Bone Loss After Pediatric Hematopoietic Cell Transplantation
NCT00101790	PHASE1	UNKNOWN	A Pilot Trial of Intravenous (IV) Pamidronate for Low Back Pain
NCT00124605	PHASE1	COMPLETED	Arsenic Trioxide and Pamidronate in Treating Patients With Advanced Solid Tumors or Multiple Myeloma
NCT00548288	PHASE1	UNKNOWN	Effect of Local Pamidronate in Preventing Bone Loss After Total Hip Arthroplasty
NCT02544659	PHASE1	COMPLETED	Efficacy of Bisphosphonates in Patients With Synovitis, Acne, Pustulosis, Hyperostosis, and Osteitis (SAPHO) Syndrome
NCT04725422	Not specified	RECRUITING	CHronic Nonbacterial Osteomyelitis International Registry
NCT00262392	Not specified	WITHDRAWN	Study of Pamidronate for the Prevention of Heterotopic Ossification
NCT00302627	Not specified	COMPLETED	Pamidronate, Vitamin D, and Calcium for the Bone Disease of Kidney and Heart Transplantation
NCT00655681	Not specified	COMPLETED	Prevention of Post Operative Bone Loss in Children
NCT02007915	Not specified	TERMINATED	The Use of Bisphosphonates in the Treatment of Avascular Necrosis of the Femoral Head

Clinical evidence (CIViC)

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

Pharmacology

Pharmacogenomics

No CPIC/DPWG dosing guideline, but PharmGKB curates 1 clinical and 5 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
RISEDRONIC ACID	ChEMBL + PubChem	Phase 4 (approved)	FDPS
ZOLEDRONIC 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