EU/3/16/1663

On 30 May 2016, orphan designation (EU/3/16/1663) was granted by the European Commission to Nextrasearch di Gasparetto Adolfo & C., Sas, Italy, for pentosan polysulfate sodium for the treatment of interstitial cystitis.

The sponsorship was transferred to NextraResearch S.r.l. - Italy, in May 2017

What is interstitial cystitis?

Interstitial cystitis (also known as painful bladder syndrome) is a long-term inflammatory condition of the bladder. The cause of the condition is unknown and a diagnosis is normally made after other likely causes such as infection have been ruled out. Symptoms include pain in the pelvic area, increased frequency of urination, and pain while urinating.

Interstitial cystitis is debilitating in the long term due to the associated pain and discomfort and the increased urinary frequency.

What is the estimated number of patients affected by the condition?

At the time of designation, interstitial cystitis affected approximately 2 in 10,000 people in the European Union (EU). This was equivalent to a total of around 103,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

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*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 28), Norway, Iceland and Liechtenstein. This represents a population of 513,700,000 (Eurostat 2016).

What treatments are available?

At the time of designation, no satisfactory methods for the treating interstitial cystitis were authorised in the EU. Painkillers were used to manage the pain. Pentosan polysulfate sodium was authorised in some countries around the world to relieve pain and discomfort in patients with interstitial cystitis.

How is this medicine expected to work?

It is thought that in interstitial cystitis defects may occur in the protective layer of molecules called glycosaminoglycans (or GAGs) in the lining of the bladder. This medicine, pentosan polysulfate sodium, is very similar to GAGs and it is expected to work by attaching to and repairing the defective GAG layer. This is expected to protect bladder cells from irritating substances in the urine, thereby improving the symptoms of the disease.

What is the stage of development of this medicine?

As pentosan polysulfate sodium is a well-known substance, the sponsor provided data in patients from the published literature to support its application for orphan designation.

At the time of submission, pentosan polysulfate sodium was authorised in several countries outside the EU for treating interstitial cystitis.

The medicine was not authorised anywhere in the EU for interstitial cystitis.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 21 April 2016 recommending the granting of this designation.

Opinions on orphan medicinal product designations are based on the following three criteria:

• the seriousness of the condition;
• the existence of alternative methods of diagnosis, prevention or treatment;
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) or insufficient returns on investment.

Designated orphan medicinal products are products that are still under investigation and are considered for orphan designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.