EU/3/13/1146

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Orphan designation

On 19 June 2013, orphan designation (EU/3/13/1146) was granted by the European Commission to Sucampo Pharma Europe Ltd, United Kingdom, for unoprostone isopropyl for the treatment of retinitis pigmentosa.

What is retinitis pigmentosa?

Retinitis pigmentosa is a group of hereditary diseases of the eye that lead to progressive loss of sight. In patients with retinitis pigmentosa, cells in the retina (the light-sensitive surface at the back of the eye) become damaged and eventually die.

Retinitis pigmentosa is a long-term debilitating disease because it causes the patient’s sight to get worse, eventually leading to blindness.

What is the estimated number of patients?

At the time of designation, retinitis pigmentosa affected less than 3.7 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 188,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).


*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 27), Norway, Iceland and Liechtenstein. This represents a population of 509,000,000 (Eurostat 2013).

What treatments are available?

At the time of designation, no satisfactory methods were authorised in the EU for treating retinitis pigmentosa. Patients with the condition were given sunglasses to slow down the damage to the retina, genetic counselling (discussion of the risks of passing the condition on to children) and general support.

How is this medicine expected to work?

Unoprostone isopropyl is a medicine authorised outside Europe for other eye disorders. The exact way in which unoprostone isopropyl works in retinitis pigmentosa is not known, although the medicine is known to affect potassium and chloride channels, which are pores through which the essential substances potassium and chloride move out of cells, and which are widely found in the eye. 

Experiments suggest that the medicine has a number of actions that could benefit patients with the condition: suppression of the programmed cell death of the retinal cells, improvement of blood flow in the retina and improving the clearing up of retinal debris.

What is the stage of development of this medicine?

The effects of unoprostone isopropyl have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with unoprostone isopropyl in patients with retinitis pigmentosa were ongoing.

At the time of submission, unoprostone isopropyl was not authorised anywhere in the EU for retinitis pigmentosa. Orphan designation had been granted in the United States for retinitis pigmentosa.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 15 May 2013 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.

Key facts

Product details for <p>unoprostone isopropyl</p>
Active substanceunoprostone isopropyl
Medicine Name
Disease/conditionTreatment of retinitis pigmentosa
Date of decision19/06/2013
OutcomePositive
Orphan decision numberEU/3/13/1146

Review of designation

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

Sponsor’s contact details

Sucampo Pharma Europe Ltd
John Eccles House
Robert Robinson Avenue
Oxford Science Park
Oxford
OX4 4GP
United Kingdom
Tel. +44 (0)1865 338003
Fax +44 (0)1865 338104
E-mail: infoeu@sucampo.com

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases which includes a directory of patients’ organisations registered in Europe;
  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.