Orphan drug affects eyesight
Having been widely prescribed for decades, the Elmiron bladder medication has recently been found to be toxic to the retina (damage).
An article published by the American Academy of Ophthalmology in 2018 described six patients experiencing pigmentary maculopathy after chronic exposure to Elmiron (pentosan polysulfate sodium) for interstitial cystitis.
The following year, an update noted that ten other IC patients had been diagnosed with the same condition.
Complaints filed against the Elmiron laboratory are claiming that long-term use of this orphan drug – developed mainly to treat painful bladder syndrome – may cause a rare eye condition called pigmentary maculopathy.
Affecting the central portion of the retina (the macula), this type of macular degeneration can lead to blindness.
In May 2020, a lawsuit was brought against J&J subsidiary Janssen Pharmaceuticals, seeking class certification in order to set up a fund to monitor the vision of patients taking Elmiron.
Several other lawsuits have also been filed by IC patients complaining of permanent vision loss caused by this bladder medication.
On the market for two decades
Approved by the FDA in 1996, Elmiron was initially owned by Alza Corporation, which merged with Johnson & Johnson in 2001 through a $10.5 billion stock-for-stock transaction.
Since then, Janssen Pharmaceuticals has marketed Elmiron under the J&J umbrella.
However, there are allegations that this laboratory failed to provide any warnings of retinopathy or maculopathy associated with this drug, and did not advise patients to monitor their sight through regular eye examinations.
Although approved by the FDA for treating interstitial cystitis, its mechanism of action is still unknown.
Believed to serve as a buffer for the delicate bladder lining – making it less leaky and thus less inflamed and painful – this medication may control cell permeability, shielding fragile mucous membranes from irritating substances in urine.
Taken as a capsule three times a day, Elmiron (pentosan polysulfate sodium) resembles blood-thinning heparin, as pentosan is a man-made drug with weak anticoagulant effects.
An effective fibrinolytic, it strengthens the bladder lining, plugging leaks for less inflammation and pain.
Massive database study
In October 2019, a study conducted by three concerned ophthalmologists at Kaiser Permanente showed that long-term Elmiron use was indeed linked to retina damage.
Trawling through the Kaiser database of 4.3 million names, they found 140 IC patients who had taken an average of 5000 pills each during fifteen years of treatment, with 91 of them agreeing to come in for tests.
After detailed retinal imaging, they were divided into three categories: normal, possible abnormality and definite abnormality.
Among them, 22 showed clear signs of drug toxicity, with this rate rising in parallel to the amount of the drug taken: up from 11% among patients taking 500 to 1000 grams to 42% among patients taking 1500 grams or more.
The current recommendation is that patients taking Elmiron with no signs of toxicity be screened for retina damage at least once a year, while anyone with eyesight difficulties should discuss the possibility of coming off this medication with their physicians.
In addition to treating painful bladder syndrome, Elmiron is also prescribed for osteoarthritis.
Prior to this disclosure, studies under way in Australia focused on repurposing pentosan polysulfate sodium (PPS) for tackling inflammation through an injectable form of Elmiron.
There were suggestions that it has anti-inflammatory effects, and prevents pain by controlling bone marrow lesions.
Particularly prone to knee injuries, American and Australian football league players were injected with PPS twice a week for six weeks in an off-label trial, reporting significant reductions in their knee pain levels.
Other conditions under consideration for treatment with Elmiron include deep vein thrombosis, and rarer diseases like Ross River fever (viral osteoarthritis) and muco-poly-saccharidosis (MPS).
Elsewhere in the world, other trials have been exploring whether pentosan polysulfate sodium could be used to treat prostate inflammation and even psychotic disorders.
Other side effects
Until very recently, the side-effects of Elmiron were viewed as relatively mild: stomach pain, diarrhoea, nausea and bloody stools; depression, dizziness and headaches; bruising, rashes and hair loss; as well as fluid buildup causing weight gain and bloating.
However, the benefits of the medication tended to outweigh these inconveniences.
Designed for rapid self-repair, the bladder could quickly heal its natural barrier against harsh waste materials in urine, as the medication shielded specialized cells restoring damaged lining tissue.
Lacking the massive markets of more commonly used drugs, Elmiron offsets low volumes by high prices.
Its shelf price over $1,300 dollars for a month’s supply (90 capsules) drops to around $900 with its SingleCare coupon.
And with no generic alternatives available, this nonpreferred, Tier IV specialty drug probably requires pre-authorization from medical aid schemes, with patients having to co-pay up to 60% of the cost.
Bladder inflammation patients unwilling to risk blindness are now left with few alternatives for managing their condition.
However, studies show that diet may trigger IC flares, with coffee, tea, soda, alcohol and citrus juices among the most bothersome, particularly with artificial sweeteners.
Hot peppers and spicy foods are obvious hazards.
Simply eliminating such bladder irritants and increasing alkalinization with potassium citrate or baking soda may help many IC patients, particularly when a steady water intake keeps urine well diluted.
For bladder patients left with no alternative medication, the bottom line is that minor lifestyle tweaks may enhance their quality of life, at least until a new medication is launched.
But for Janssen Pharmaceuticals, its bottom line may soon reflect the financial consequences of this tragic side effect.