Elmiron, or pentosan polysulfate sodium, is an oral prescription drug used to treat discomfort or bladder pain associated with interstitial cystitis, or IC, a chronic condition that causes bladder pain and pressure. The most common side effects of Elmiron are hair loss, diarrhea and nausea. Headache, abdominal pain and upset stomach may also occur.
Janssen Pharmaceuticals manufactures Elmiron, and the U.S. Food and Drug Administration originally approved it for sale in 1996. It was approved under the Orphan Drug Act, or ODA. The ODA gives special status and incentives to sponsors, or manufacturers, of medications that treat rare diseases.
Currently, it’s the only oral drug approved to treat bladder pain and discomfort associated with IC, and it’s not available in generic. Since its release, it’s estimated that hundreds of thousands of people have used the drug, according to HealthDay. Most people who use the drug are women, and the drug is most often used long-term.
The majority of Elmiron side effects are mild, but new reports suggest the drug could be toxic to the retina, the tissue behind the eye that senses light.
How Pentosan Polysulfate Sodium Works
Researchers don’t know exactly how pentosan polysulfate sodium works, but they believe it works by protecting the bladder from irritants in urine. Elmiron doesn’t work like other pain relievers like Tylenol or non-steroidal anti-inflammatory drugs like ibuprofen, and it doesn’t work by stopping inflammation.
In people with IC, the bladder wall’s protective lining is damaged. This allows irritating substances in urine to cause inflammation in the bladder wall. Elmiron creates a barrier on the bladder wall to prevent irritation.
Does Elmiron Cure Interstitial Cystitis?
Elmiron doesn’t cure IC, but it does provide some relief from symptoms. Interstitial cystitis, or bladder pain syndrome, is a chronic condition that causes painful symptoms in the bladder. IC is common and affects between 1 to 4 million men and between 3 to 8 million women in the United States, according to the National Institute of Diabetes and Digestive and Kidney Diseases. Currently, there is no cure.
Researchers don’t know what causes IC, but certain activities, food and drinks may trigger symptoms. For example, people reported symptom flares with stress, intercourse, holding their urine or using tight undergarments.
- Pressure, mild discomfort or tenderness in the bladder, lower abdomen or pelvic area
- Urinary urgency several times a day
- Waking up several times at night to urinate
- Burning sensation or pain
- Urinating more often
- Pain that gets worse until bladder is emptied
- Pelvic floor muscle pain
- Pain during or after sexual intercourse
How Well Does It Work?
Elmiron’s manufacturers tested the drug in two clinical trials in 2,627 patients. Of these trial participants, 2,343 were women, 262 were men and 22 were unknown. After three months in the second trial, 52 percent of the patients, or 1,307, had dropped out or were ineligible for analysis, according to the medication insert.
The results of these studies showed that Elmiron relieved pain in many patients. A majority of patients who had a response demonstrated pain relief by 3 months; however, a small percent of patients took up to 6 months to demonstrate pain relief.
But another study done on a smaller number of patients by G.R. Sant and colleagues published in The Journal of Urology found that oral pentosan polysulfate sodium didn’t provide much benefit to the majority of IC patients.
Elmiron has a few common side effects, and most of them are mild. Because it’s a mild blood thinner, it may also cause bruising. Call your doctor right away if these side effects persist or bother you or if you have blood in your stool.
- Abnormal liver function tests
- Blood in the stool
- Hair loss
- Upset stomach
Some people reported pigmentary changes in the retina, also called pigmentary maculopathy, with long-term use of Elmiron. Researchers don’t know what causes it, but it can cause problems with reading, blurred vision and difficulty adjusting to low lighting. Call your doctor if you notice changes in your vision while taking this medicine.
Dosage, Instructions and Precautions
The recommended daily dose for Elmiron is one 100 mg capsule by mouth three times a day for a total of 300 mg daily. The capsule should be taken with water at least one hour before meals or two hours after meals.
After three months of taking Elmiron, the patient should contact their health care provider for an evaluation of how well the drug is working. If there is no improvement in IC symptoms and there are no serious side effects, the health care provider may recommend another three months of treatment.
Elmiron is a weak anticoagulant. This means it works like a blood thinner and makes it harder for blood to clot. Its strength is about one-fifteenth the strength of heparin. Rectal hemorrhage, gum hemorrhage and bruising have been reported.
Patients about to have surgery, who have symptoms of bleeding or who are taking other drugs that may increase bleeding — blood thinners, aspirin, or non-steroidal anti-inflammatory drugs — should tell their health care providers right away.
Some patients experienced mildly elevated liver enzymes that occurred three to 12 months after starting treatment. Researchers haven’t tested Elmiron in people with liver insufficiency, and people with liver problems should use the drug with caution.
Pregnancy and Breastfeeding
Elmiron should be used in pregnancy only if clearly needed, according to the drug’s label.
Reproduction studies in mice and rats did not reveal evidence of impaired fertility or harm to the fetus from Elmiron. However, adequate and well-controlled studies have not been performed in pregnant women, and animal studies are not always predictive of human response.
Nursing women should also exercise caution. Many drugs are excreted in human milk, and it is not known whether Elmiron can pass through breast milk.
Elmiron’s medication insert doesn’t list any drug interactions. But patients should tell their health care provider about all drugs, vitamins and any herbal supplements they are taking — especially any medication that may increase bleeding risk.
Lawsuits for Eye Damage
Elmiron’s medication insert didn’t list any warnings about eye damage or an increased risk of eye disease until Janssen added a warning about pigmentary maculopathy in June 2020, according to lawsuits.
One of the first case reports appeared in the Journal of Urology in 2018. Dr. William A. Pearce and colleagues wrote that long-term use of the drug led to “injury to the retina and the underlying retinal pigment epithelium,” according to the IC Network. Since that report, additional studies have been done to determine whether this is a true effect of the drug. Results have been mixed; however, there is suggestion that long-term use of Elmiron can cause eye damage. Canadian researcher Derrick Wang and his colleagues recommend all patients have a thorough eye examination before starting Elmiron and once a year after that to check for any changes.
In 2019, Drs. Rachel M. Huckfeldt and Demetrios G. Vavvas published a case report in Ophthalmic Surgery, Lasers and Imaging Retina of a patient whose pentosan polysulfate sodium-related maculopathy continued to get worse 6 years after stopping the drug.
Please seek the advice of a medical professional before making health care decisions.