PENTOSAN POLYSULFATE

Description
Sources: http://www.accessdata.fda.gov/drugsatfda_docs/label/2008/020193s009lbl.pdf | https://www.ncbi.nlm.nih.gov/pubmed/17071728

Pentosan polysulfate sodium (brand name ELMIRON) is a low molecular weight heparin-like compound. It has anticoagulant and fibrinolytic effects and is indicated for the relief of bladder pain or discomfort associated with interstitial cystitis. The mechanism of action of pentosan polysulfate sodium in interstitial cystitis is not known but was discovered, that it t binds Fibroblast growth factors (FGFs) as well as other heparin-binding growth factors.

Approval Year

Targets

Primary Target	Pharmacology	Condition	Potency
Fibroblast growth factor receptor 6 Target ID: CHEMBL2095217 Sources: https://www.ncbi.nlm.nih.gov/pubmed/8946244 \| https://www.ncbi.nlm.nih.gov/pubmed/17071728	Antagonist 2849

Conditions

Condition	Modality	Targets	Highest Phase	Product
Interstitial cystitis 6 Sources: http://www.accessdata.fda.gov/drugsatfda_docs/label/2008/020193s009lbl.pdf	Palliative		Approved 8583	ELMIRON Approved Use ELMIRON® (pentosan polysulfate sodium) is indicated for the relief of bladder pain or discomfort associated with interstitial cystitis. Launch Date 1996

C_max

Value	Dose	Analyte	Population
68.7 ng/mL OTHER GOV'T https://www.ema.europa.eu/en/documents/assessment-report/elmiron-epar-public-assessment-report_en.pdf#page=47 \| https://pubmed.ncbi.nlm.nih.gov/9815633/	400 mg/m² 3 times / day multiple, oral dose: 400 mg/m² route of administration: Oral experiment type: MULTIPLE co-administered:	PENTOSAN POLYSULFATE plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: UNKNOWN
462 ng/mL OTHER GOV'T https://www.ema.europa.eu/en/documents/assessment-report/elmiron-epar-public-assessment-report_en.pdf#page=47 \| https://pubmed.ncbi.nlm.nih.gov/9815633/	400 mg/m² 3 times / day multiple, oral dose: 400 mg/m² route of administration: Oral experiment type: MULTIPLE co-administered:	PENTOSAN POLYSULFATE plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: UNKNOWN
250 ng × eq/mL OTHER GOV'T https://www.ema.europa.eu/en/documents/assessment-report/elmiron-epar-public-assessment-report_en.pdf#page=47 \| https://pubmed.ncbi.nlm.nih.gov/16278190/	300 mg single, oral dose: 300 mg route of administration: Oral experiment type: SINGLE co-administered:	PENTOSAN POLYSULFATE plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE food status: FASTED
358 ng × eq/mL OTHER GOV'T https://www.ema.europa.eu/en/documents/assessment-report/elmiron-epar-public-assessment-report_en.pdf#page=47 \| https://pubmed.ncbi.nlm.nih.gov/16278190/	450 mg single, oral dose: 450 mg route of administration: Oral experiment type: SINGLE co-administered:	PENTOSAN POLYSULFATE plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE food status: FASTED

AUC

Value	Dose	Analyte	Population
987.5 ng × h/mL OTHER GOV'T https://www.ema.europa.eu/en/documents/assessment-report/elmiron-epar-public-assessment-report_en.pdf#page=47 \| https://pubmed.ncbi.nlm.nih.gov/9815633/	400 mg/m² 3 times / day multiple, oral dose: 400 mg/m² route of administration: Oral experiment type: MULTIPLE co-administered:	PENTOSAN POLYSULFATE plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: UNKNOWN
6660 ng × h/mL OTHER GOV'T https://www.ema.europa.eu/en/documents/assessment-report/elmiron-epar-public-assessment-report_en.pdf#page=47 \| https://pubmed.ncbi.nlm.nih.gov/9815633/	400 mg/m² 3 times / day multiple, oral dose: 400 mg/m² route of administration: Oral experiment type: MULTIPLE co-administered:	PENTOSAN POLYSULFATE plasma	Homo sapiens population: UNHEALTHY age: ADULT sex: FEMALE / MALE food status: UNKNOWN
21 ng × eq × h/mL OTHER GOV'T https://www.ema.europa.eu/en/documents/assessment-report/elmiron-epar-public-assessment-report_en.pdf#page=47 \| https://pubmed.ncbi.nlm.nih.gov/16278190/	300 mg single, oral dose: 300 mg route of administration: Oral experiment type: SINGLE co-administered:	PENTOSAN POLYSULFATE plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE food status: FASTED
33.8 ng × eq × h/mL OTHER GOV'T https://www.ema.europa.eu/en/documents/assessment-report/elmiron-epar-public-assessment-report_en.pdf#page=47 \| https://pubmed.ncbi.nlm.nih.gov/16278190/	450 mg single, oral dose: 450 mg route of administration: Oral experiment type: SINGLE co-administered:	PENTOSAN POLYSULFATE plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE food status: FASTED

T_1/2

Value	Dose	Co-administered	Analyte	Population
26.5 h OTHER GOV'T https://www.ema.europa.eu/en/documents/assessment-report/elmiron-epar-public-assessment-report_en.pdf#page=47 \| https://pubmed.ncbi.nlm.nih.gov/16278190/	300 mg single, oral dose: 300 mg route of administration: Oral experiment type: SINGLE co-administered:		PENTOSAN POLYSULFATE plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE food status: FASTED
19.5 h OTHER GOV'T https://www.ema.europa.eu/en/documents/assessment-report/elmiron-epar-public-assessment-report_en.pdf#page=47 \| https://pubmed.ncbi.nlm.nih.gov/16278190/	450 mg single, oral dose: 450 mg route of administration: Oral experiment type: SINGLE co-administered:		PENTOSAN POLYSULFATE plasma	Homo sapiens population: HEALTHY age: ADULT sex: FEMALE food status: FASTED

Doses

Dose	Population	Adverse events
1500 mg single, oral Highest studied dose Dose: 1500 mg Route: oral Route: single Dose: 1500 mg Sources: https://pubmed.ncbi.nlm.nih.gov/10192753/	healthy, ADULT Health Status: healthy Age Group: ADULT Sex: M Food Status: FASTED Sources: https://pubmed.ncbi.nlm.nih.gov/10192753/	Sources: https://pubmed.ncbi.nlm.nih.gov/10192753/
400 mg/m2 3 times / day multiple, oral Highest studied dose Dose: 400 mg/m2, 3 times / day Route: oral Route: multiple Dose: 400 mg/m2, 3 times / day Sources: https://pubmed.ncbi.nlm.nih.gov/9815633/	unhealthy, ADULT Health Status: unhealthy Age Group: ADULT Sex: M Food Status: UNKNOWN Sources: https://pubmed.ncbi.nlm.nih.gov/9815633/	DLT: Proctitis... Disc. AE: Diarrhea, GI bleeding... Other AEs: Hematuria, Gastritis... Dose limiting toxicities: Proctitis (grade 3-4, 18%) AEs leading to discontinuation/dose reduction: Diarrhea (grade 4, 27%) GI bleeding (grade 1-2, 27%) Other AEs: Hematuria (grade 1, 9%) Gastritis (4.5%) Sources: https://pubmed.ncbi.nlm.nih.gov/9815633/

AEs

AE	Significance	Dose	Population
Gastritis	4.5%	400 mg/m2 3 times / day multiple, oral Highest studied dose Dose: 400 mg/m2, 3 times / day Route: oral Route: multiple Dose: 400 mg/m2, 3 times / day Sources: https://pubmed.ncbi.nlm.nih.gov/9815633/	unhealthy, ADULT Health Status: unhealthy Age Group: ADULT Sex: M Food Status: UNKNOWN Sources: https://pubmed.ncbi.nlm.nih.gov/9815633/
Hematuria	grade 1, 9%	400 mg/m2 3 times / day multiple, oral Highest studied dose Dose: 400 mg/m2, 3 times / day Route: oral Route: multiple Dose: 400 mg/m2, 3 times / day Sources: https://pubmed.ncbi.nlm.nih.gov/9815633/	unhealthy, ADULT Health Status: unhealthy Age Group: ADULT Sex: M Food Status: UNKNOWN Sources: https://pubmed.ncbi.nlm.nih.gov/9815633/
GI bleeding	grade 1-2, 27% Disc. AE	400 mg/m2 3 times / day multiple, oral Highest studied dose Dose: 400 mg/m2, 3 times / day Route: oral Route: multiple Dose: 400 mg/m2, 3 times / day Sources: https://pubmed.ncbi.nlm.nih.gov/9815633/	unhealthy, ADULT Health Status: unhealthy Age Group: ADULT Sex: M Food Status: UNKNOWN Sources: https://pubmed.ncbi.nlm.nih.gov/9815633/
Proctitis	grade 3-4, 18% DLT, Disc. AE	400 mg/m2 3 times / day multiple, oral Highest studied dose Dose: 400 mg/m2, 3 times / day Route: oral Route: multiple Dose: 400 mg/m2, 3 times / day Sources: https://pubmed.ncbi.nlm.nih.gov/9815633/	unhealthy, ADULT Health Status: unhealthy Age Group: ADULT Sex: M Food Status: UNKNOWN Sources: https://pubmed.ncbi.nlm.nih.gov/9815633/
Diarrhea	grade 4, 27% Disc. AE	400 mg/m2 3 times / day multiple, oral Highest studied dose Dose: 400 mg/m2, 3 times / day Route: oral Route: multiple Dose: 400 mg/m2, 3 times / day Sources: https://pubmed.ncbi.nlm.nih.gov/9815633/	unhealthy, ADULT Health Status: unhealthy Age Group: ADULT Sex: M Food Status: UNKNOWN Sources: https://pubmed.ncbi.nlm.nih.gov/9815633/

PubMed

Title	Date	PubMed
Successful drug development despite adverse preclinical findings part 1: processes to address issues and most important findings.	2010-12	22272031
Sulfated dextrans enhance in vitro amplification of bovine spongiform encephalopathy PrP(Sc) and enable ultrasensitive detection of bovine PrP(Sc).	2010-10-04	20957174
The role of hypoxia in 2-butoxyethanol-induced hemangiosarcoma.	2010-01	19812364
Early identification of interstitial cystitis may avoid unnecessary hysterectomy.	2009-10-02	19793476
Patients with chronic pelvic pain: endometriosis or interstitial cystitis/painful bladder syndrome?	2007-09-01	17761077
Ketamine-associated ulcerative cystitis: a new clinical entity.	2007-05	17482909
Pentosan polysulfate (Elmiron): in vitro effects on prostate cancer cells regarding cell growth and vascular endothelial growth factor production.	2006-11	17071199
Molecular size affects urine excretion of pentosan polysulfate.	2006-03	16469641
Pentosan polysulfate: a review of its use in the relief of bladder pain or discomfort in interstitial cystitis.	2006	16706553
Metabolism of [3H]pentosan polysulfate sodium (PPS) in healthy human volunteers.	2005-08	16278190
Cyclophosphamide induced hemorrhagic cystitis successfully treated with pentosanpolysulphate.	2005-01	15592042
NTP technical report on the toxicology and carcinogenesis studies of Elmiron (Cas No. 37319-17-8) in F344/N rats and B6C3F1 mice (Gavage Studies).	2004-05	15213766
Toxicity and carcinogenicity of Elmiron in F344/N rats and B6C3F1 mice following 2 years of gavage administration.	2003-12	14508637
Lysosomal-storage disorder induced by elmiron following 90-days gavage administration in rats and mice.	2002-04-13	11950161
Effect of pentosan polysulfate therapy on intravesical potassium sensitivity.	2002-03	11880064
Osteoarthritis, genetic and molecular mechanisms.	2002	12014849
Pentosan polysulfate decreases proliferation and extracellular matrix deposition by vascular smooth muscle cells isolated from failed hemodialysis access grafts.	2000-08	10968687
Pentosan induced cerebral sagittal sinus thrombosis: a variant of heparin induced thrombocytopenia.	1998-12	9817346
Pentosan polysulfate-induced thrombocytopenia: a case diagnosed with an ELISA test used for heparin-induced thrombocytopenia.	1996-07	8695727
Effect of sodium pentosan polysulphate on the thrombogenicity of prothrombin complex concentrates.	1992-07-01	1279834
Trans-repressor activity of nuclear glycosaminoglycans on Fos and Jun/AP-1 oncoprotein-mediated transcription.	1992-01	1730747
Thrombocytopenia with or without thrombosis after pentosan polysulfate treatment.	1990-11-01	1706895
[Myocardial infarction and massive biventricular thrombosis during thrombocytopenia induced by pentosan polysulfate and heparin].	1990-01	1689564
[Disseminated coronary thrombosis and thrombopenia induced by pentosan polysulfate].	1988-07	2461180
[Myocardial infarct complicating thrombopenia induced by pentosan polysulfate].	1988-04	2453137
[Thrombopenia and fatal intracerebral hemorrhage caused by pentosan polysulfate].	1988-01-30	2450343

Patents

Sample Use Guides

In Vivo Use Guide

The recommended dose of ELMIRON® (pentosan polysulfate sodium, capsules) is 300 mg/day taken as one 100 mg capsule orally three times daily. The capsules should be taken with water at least 1 hour before meals or 2 hours after meals. Patients receiving ELMIRON® should be reassessed after 3 months. If improvement has not occurred and if limiting adverse events are not present, ELMIRON® may be continued for another 3 months.

Route of Administration: Oral

In Vitro Use Guide

Effect of pentosan polysulfate (PPS) on p38, ERK1/2 and JNK MAPK activation was studied. Canine chondrocytes were plated onto 6-well culture plates in serum free DMEM for 24 hr. Cells were pre-treated with 100 µg/ml of PPS for 1 hr and then incubated with 10 ng/ml of recombinant human (rh) IL-1β for 5, 15, 30, 60 or 240 min. It was shown, that phosphorylation of p38 and ERK were inhibited by 100 mg/ml of PPS, while JNK remained constant despite PPS pretreatment prior to rhIL-1β stimulation. Effect of PPS on NF-κB translocation induced by rhIL- 1β: Under control conditions, a diffuse cytoplasmic staining was seen and the average percentage of NF-κB positive nuclei cells was 9.5%. On the other hand, 78.2% of chondrocytes incubated with rhIL- 1β had a nuclear staining pattern, indicating NF-κB nuclear translocation. Preincubation with 10 and 100 µg/ml PPS reduced NF-κB translocation rate.

Substance Class	Polymer Created by `admin` on `Mon Mar 31 22:59:32 GMT 2025` Edited by `admin` on `Mon Mar 31 22:59:32 GMT 2025`
Record UNII	F59P8B75R4
Record Status	`Validated (UNII)`
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Name

Type

Language

PENTOSAN POLYSULFATE

Common Name

English

PENTOSAN POLYSULFATE [MI]

Preferred Name

English

XYLAN, HYDROGEN SULFATE

Common Name

English

(4-O-METHYL-.ALPHA.-D-GLUCURONO)-(1->2)-(1->4)-.BETA.-D-XYLOPYRANAN, HYDROGEN SULFATE

Common Name

English

PENTOSAN POLYSULPHATE

Common Name

English

4-O-METHYL-.ALPHA.-D-GLUCURONO-.BETA.-D-XYLAN, HYDROGEN SULFATE

Common Name

English

XYLAN, HYDROGEN SULPHATE

Common Name

English

4-O-METHYL-.ALPHA.-D-GLUCURONO-.BETA.-D-XYLAN, HYDROGEN SULPHATE

Common Name

English

(4-O-METHYL-.ALPHA.-D-GLUCURONO)-(1->2)-(1->4)-.BETA.-D-XYLOPYRANAN, HYDROGEN SULPHATE

Common Name

English

Pentosan polysulfate [WHO-DD]

Common Name

English

Classification Tree Code System Code

NDF-RT

N0000175837