Date of Revision: July 11, 2008

Submission Control No: 121174

*Registered trademark of IVAX Research, LLC. Used under license. (c) 2008 JANSSEN-ORTHO Inc.

Table of Contents

PART I: HEALTH PROFESSIONAL INFORMATION 3

SUMMARY PRODUCT INFORMATION 3 INDICATIONS AND CLINICAL USE 3 CONTRAINDICATIONS 3 WARNINGS AND PRECAUTIONS 4 ADVERSE REACTIONS 5 DRUG INTERACTIONS 7 DOSAGE AND ADMINISTRATION 7 OVERDOSAGE 7 ACTION AND CLINICAL PHARMACOLOGY 8 STORAGE AND STABILITY 9 DOSAGE FORMS, COMPOSITION AND PACKAGING 9

PART II: SCIENTIFIC INFORMATION 10

PHARMACEUTICAL INFORMATION 10 CLINICAL TRIALS 10 DETAILED PHARMACOLOGY 11 TOXICOLOGY 12 REFERENCES 14

PART III: CONSUMER INFORMATION. 17

Pr ELMIRON * pentosan polysulfate sodium Capsules 100 mg Glycosaminoglycan Substitute

Route of Administration Dosage Form / Strength Clinically Relevant Nonmedicinal Ingredients
oral capsules 100 mg For a complete listing, see DOSAGE FORMS, COMPOSITION AND PACKAGING section.

PART I: HEALTH PROFESSIONAL INFORMATION SUMMARY PRODUCT INFORMATION

INDICATIONS AND CLINICAL USE

ELMIRON (pentosan polysulfate sodium) is indicated for the initial and maintenance treatment of interstitial cystitis.

Pediatrics (< 18 years of age):

Safety and effectiveness in children and adolescents below the age of 18 years have not been established.

CONTRAINDICATIONS

Patients who are hypersensitive to this drug or to any ingredient in the formulation or component of the container. For a complete listing, see the DOSAGE FORMS, COMPOSITION AND PACKAGING section of the Product Monograph.

WARNINGS AND PRECAUTIONS

General

ELMIRON is a weak anticoagulant (only 1/15 the activity of heparin) and has been used in prevention of thrombotic disease.

Carcinogenesis and Mutagenesis

Long-term carcinogenicity studies in rats showed no evidence of carcinogenic potential at exposures up to 60 times the maximum recommended human dose (MRHD) on a mg/kg basis. In a 2-year carcinogenicity study in mice, there was an increase in hemangiosarcomas in male mice and hepatocellular neoplasms in male and female mice at a dose approximately 117 times the MRHD on a mg/kg basis. No mutagenic activity has been observed. See Product Monograph Part II: Mutagenesis and Carcinogenesis sections for discussion on animal data.

Hematologic

A small number of bleeding complications of ecchymosis, epistaxis, and gum hemorrhage have been reported (see ADVERSE REACTIONS). At a daily dose of 300 mg (n = 128), rectal hemorrhage was reported as an adverse event in 6.3% of patients. Patients at increased hemorrhagic risk due to diseases such as ulcerative GI lesions, aneurysms, internal or external hemorrhoids, thrombocytopenia, hemophilia, polyps or diverticulae should also be evaluated carefully if they are to receive ELMIRON.

Hepatic

Pentosan polysulfate sodium is desulfated by both the liver and the spleen. The extent to which hepatic insufficiency or splenic disorders may increase the bioavailability of the parent or active metabolites of pentosan polysulfate sodium is not known. Caution should be exercised when using ELMIRON in these patients. Mildly (<2.5 x normal) elevated transaminase, alkaline phosphatase, g-glutamyl transpeptidase, and lactic dehydrogenase occurred in 1.2% of patients. The increases usually appeared 3 to 12 months after the start of ELMIRON therapy, and were not associated with jaundice or other clinical signs or symptoms. These abnormalities are usually transient, may remain essentially unchanged, or may rarely progress with continued use.

Peri-Operative Considerations

Patients undergoing invasive procedures, having signs or symptoms of underlying coagulopathy or who are otherwise at increased risk of bleeding (due to other therapies such as coumarin anticoagulants e.g. warfarin, heparin, tPA, streptokinase, high-dose aspirin, or nonsteroidal anti- inflammatories) should be evaluated for hemorrhagic risk.

Reproduction

Reproductive studies performed in the rat had no effect on fertility. The effect of pentosan polysulfate sodium on spermatogenesis has not been investigated. See Product Monograph Part II: Teratology section for discussion on animal data.

Special Populations

Pregnant Women:

There are no adequate and well-controlled studies in pregnant women. Therefore, this drug should be used during pregnancy only if the potential benefit clearly exceeds the potential risk.

Nursing Women:

It is not known if ELMIRON is excreted in human milk. Many drugs are excreted in human milk; therefore, caution should be exercised when ELMIRON is administered to a nursing mother.

Pediatrics (< 18 years of age):

Safety and effectiveness in children and adolescents below the age of 18 years have not been established. This drug should be kept out of the reach of children.

ADVERSE REACTIONS

Adverse Drug Reaction Overview

ELMIRON is usually well tolerated. Reported adverse reactions are infrequent and usually do not require discontinuation of treatment. The most common reactions are gastrointestinal, hematologic or dermatologic (see WARNINGS AND PRECAUTIONS). Adverse events reported are summarized in Tables 1.1 and 1.2.

Table 1.1 Low Frequency (#3%) Adverse Events Reported in Patients Treated with ELMIRON

BODY SYSTEM ADVERSE EVENT
Body as a Whole Headache
Digestive GI discomfort Diarrhea Nausea
Skin and Appendages Alopecia Rash

Table 1.2 Uncommon (#1%) Adverse Events Reported in Patients Treated with ELMIRON

BODY SYSTEM ADVERSE EVENT
Body as a Whole Malaise Pelvic pain
Digestive Liver function abnormalities Vomiting Mouth ulcer Colitis Esophagitis Gastritis
Hematologic Anemia Ecchymosis Prothrombin decrease Thrombocytopenia Retinal hemorrhage
Hypersensitive Reactions Allergic reaction Photosensitivity
Metabolic Weight gain Weight loss Edema
Musculoskeletal Myalgia Arthralgia
Neurologic Dizziness Paresthesia Insomnia
Respiratory Sinusitis
Skin and Appendages Sweating
Urogenital Urgency Urinary tract infection Urethritis

Rare events (single occurrence only in over 1,000 patients with interstitial cystitis): gastritis, leukopenia, depression, rhinitis, lacrimation, angina pectoris, chronic myelogenous leukemia, prostate cancer, loss of appetite, subarachnoid hemorrhage, epistaxis, gum hemorrhage, menorrhagia and hematuria.

Post-Market Adverse Drug Reactions

Rectal hemorrhage: ELMIRON was evaluated in a randomized, double-blind, parallel-group, Phase 4 study conducted in 380 patients with interstitial cystitis dosed for 32 weeks. At a daily dose of 300 mg (n = 128), rectal hemorrhage was reported as an adverse event in 6.3% of patients. The severity of the events was described as "mild" in most patients. Patients in that study who were administered ELMIRON 900 mg daily, a dose higher than the approved dose, experienced a higher incidence of rectal hemorrhage, 15%. Liver Function Abnormality: A randomized, double-blind, parallel-group, Phase 2 study was conducted in 100 men (51 ELMIRON and 49 placebo) dosed for 16 weeks. At a daily dose of 900 mg, a dose higher than the approved dose, elevated liver function tests were reported as an adverse event in 11.8% (n = 6) of ELMIRON treated patients and 2% (n = 1) of placebo-treated patients.

DRUG INTERACTIONS

Drug-Drug Interactions

In a study in which healthy subjects (24 completed) received pentosan polysulfate sodium 100 mg or placebo every 8 hours for 7 days, and were titrated with warfarin to an INR of 1.4 to 1.8, the pharmacokinetic parameters of R-warfarin and S-warfarin were similar in the absence or presence of pentosan polysulfate sodium. Prothrombin time, partial thromboplastin time and INR for warfarin + placebo and warfarin + pentosan polysulfate sodium were comparable. See also WARNINGS AND PRECAUTIONS, Peri-Operative Considerations. Drug interactions with other anticoagulant drugs have not been studies. Care should be taken when administering ELMIRON to patients receiving anticoagulant drugs such as warfarin, heparin, tPA, streptokinase, high-dose aspirin, and nonsteroidal anti- inflammatory drugs.

DOSAGE AND ADMINISTRATION

Dosing Considerations

The capsules should be taken with water at least 1 hour before meals or 2 hours after meals.

Recommended Dose

The recommended dose of ELMIRON is 300 mg/day taken as one 100 mg capsule orally three times daily. Some patients with interstitial cystitis may require 6 to 8 weeks of therapy with ELMIRON to achieve relief of symptoms. Long-term continuation of ELMIRON therapy is necessary for persistent therapeutic effect.

OVERDOSAGE

Overdose has not been reported. Based upon the pharmacodynamics of the drug, toxicity is likely to be reflected as anticoagulation, bleeding, thrombocytopenia, liver function abnormalities, and gastric distress. (See ACTION AND CLINICAL PHARMACOLOGY and WARNINGS AND PRECAUTIONS sections). At a daily dose of 900 mg for 32 weeks (n = 127) in a clinical trial, rectal hemorrhage was reported as an adverse event in 15% of patients. At a daily dose of ELMIRON 900 mg for 16 weeks in a clinical trial that enrolled 51 patients in the ELMIRON group and 49 in the placebo group, elevated liver function tests were reported as an adverse event in 11.8% of patients in the ELMIRON group and 2% of patients in the placebo group. In the event of acute overdosage, the patient should be given gastric lavage if possible, carefully observed and given symptomatic and supportive treatment.

ACTION AND CLINICAL PHARMACOLOGY

Mechanism of Action

ELMIRON is orally bioavailable pentosan polysulfate sodium. Its mechanism of action is thought to be adherence to the bladder surface supplementing the defective natural glycosaminoglycan layer. It is hypothesized that this action ameliorates the symptoms of interstitial cystitis.

Pharmacodynamics

ELMIRON is intended for the treatment of interstitial cystitis. In interstitial cystitis patients, a deficient or defective bladder protective glycosaminoglycan layer allows diffusion of irritating components in urine through to the underlying bladder wall. The resultant inflammatory response in the bladder wall produces the symptoms of interstitial cystitis. Definitive proof for this is not available. In addition to its action as a glycosaminoglycan replacement in the bladder, pentosan polysulfate sodium has weak anticoagulant effect, fibrinolytic effect, a lipolytic effect and anti-inflammatory actions.

Absorption

In a clinical pharmacology study in which healthy female volunteers received a single oral 300 mg or 450 mg dose of pentosan polysulfate sodium containing radiolabeled drug under fasted conditions, mean maximal levels of plasma radioactivity were seen approximately 2 hours after dosing. Based on urinary excretion of radioactivity, a mean of approximately 6% of a radiolabeled oral dose of pentosan polysulfate sodium is absored and reaches the systemic circulation. In clinical trials, ELMIRON, was administered with water 1 hour before or 2 hours after meals; the effect of food on absorption of pentosan polysulfate sodium is not known.

Distribution

Parenteral radiolabelled studies in animals indicate significant distribution to the uroepithelium of the genitourinary tract with lesser amounts found in the liver, spleen, lung, skin, periosteum and bone marrow. Erythrocyte penetration is very low.

Metabolism

The absorbed fraction of a dose of pentosan polysulfate sodium is metabolized by partial desulfation in the liver and spleen, and by partial depolymerization in the kidney to a large number of metabolites. Both the desulfation and depolymerization can be saturated with continued dosing.

Excretion

Following oral administration of a 300 or 450 mg dose of pentosan polysulfate sodium containing radiolabeled drug to groups of healthy subjects, plasma radioactivity declined with mean half-lives of 27 and 20 hours, respectively. Most of an orally administered dose of pentosan polysulfate sodium (mean 84%) is excreted in feces as unchanged drug. A mean of 6% of an oral dose is excreted in the urine, mostly as desulfated and depolymerized metabolites. Only a small fraction of the administered dose (mean 0.14%) is recovered as intact drug.

Special Populations

No data are available in geriatric patients or in patients with hepatic or renal impairment. See also WARNINGS AND PRECAUTIONS, Hepatic.

STORAGE AND STABILITY

Store at controlled room temperature (15E to 30E C).

DOSAGE FORMS, COMPOSITION AND PACKAGING

ELMIRON is supplied in white opaque hard gelatin capsules imprinted "BNP7600" containing 100 mg pentosan polysulfate sodium. Each capsule of ELMIRON contains 100 mg pentosan polysulfate sodium and the following inactive ingredients: gelatin, magnesium stearate, microcrystalline cellulose, and titanium dioxide. Available in bottles of 100 capsules.