Bene Arzneimittel GmBH, a german pharmaceutical company, announced in late December 2015 that Pentosan polysulphate (aka Elmiron) has been officially registered as an orphan drug for the treatment of interstitial cystitis by the European Medicines Agency, EMA. “Receiving orphan drug status for pentosan polysulphate in the European Union is a great success for bene”, says CEO Dr Guenter Auerbach.
Orphan drug status offers several benefits for pharmaceutical companies, usually in the form of reduced fees and exclusive drug sales for a period of time. What I find baffling, however, is their classification of IC as a rare disease. Studies in the USA have estimated that 3 to 8 million women and 1 to 4 million men have symptoms of IC making this far from rare. In Europe, however, the term “interstitial cystitis” is only applied to patients who are diagnosed with Hunner’s lesions in the bladder. Patients with traditional IC symptoms (frequency, urgency, pressure, pain as the bladder fills) who do not have lesions are diagnosed with bladder pain syndrome (BPS) instead. Thus, it appears that they have achieved orphan status by focusing on this much smaller subset of IC patients.
That said, very few (if any) clinicians in the USA have suggested that pentosan polysulfate is useful in the treatment of Hunner’s lesions. The AUA treatment guidelines for IC/BPS specifically note that if lesions are present, they are generally treated with fulguration, laser therapy or with an injection of triamcinolone into the center of the lesion.
So what does this mean? It’s confusing. My concern is that this could block the incentive for generic manufacturers from entering the pentosan market. Given the rising costs of Elmiron in the USA, patients are desperately awaiting a cheaper, generic formulation. This loss of marketshare could be a powerful deterrance.