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Orphan Drug Status for Pentosan May Deter Lower Price Generics

costofmedicineBene 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.

Jill O.

By | 2017-01-31T08:58:00+00:00 January 8th, 2016|Industry News, Interstitial Cystitis Network Blog, News|Comments Off on Orphan Drug Status for Pentosan May Deter Lower Price Generics

About the Author:

My Google Profile+ Jill Heidi Osborne is the president and founder of the Interstitial Cystitis Network, a health education company dedicated to interstitial cystitis, bladder pain syndrome and other pelvic pain disorders. As the editor and lead author of the ICN and the IC Optimist magazine, Jill is proud of the academic recognition that her website has achieved. The University of London rated the ICN as the top IC website for accuracy, credibility, readability and quality. (Int Urogynecol J - April 2013). Harvard Medical School rated both Medscape and the ICN as the top two websites dedicated to IC. (Urology - Sept 11). Jill currently serves on the Congressionally Directed Medical Research Panel (US Army) where she collaborates with researchers to evaluate new IC research studies for possible funding. Jill has conducted and/or collaborates on a variety of IC research studies on new therapeutics, pain care, sexuality, the use of medical marijuana, menopause and the cost of treatments, shining a light on issues that influence patient quality of life. An IC support group leader and national spokesperson for the past 20 years, she has represented the IC community on radio, TV shows, at medical conferences. She has written hundreds of articles on IC and its related conditions. With a Bachelors Degree in Pharmacology and a Masters in Psychology, Jill was named Presidential Management Intern (aka Fellowship) while in graduate school. (She was unable to earn her PhD due to the onset of her IC.) She spends the majority of her time providing WELLNESS COACHING for patients in need and developing new, internet based educational and support tools for IC patients, including the “Living with IC” video series currently on YouTube and the ICN Food List smartphone app! Jill was diagnosed with IC at the age of 32 but first showed symptoms at the age of 12.