EU/3/07/500

On 29 November 2007, orphan designation (EU/3/07/500) was granted by the European Commission to Fovea Pharmaceuticals SA, France, for recombinant human rod-derived cone viability factor for the treatment of treatment of retinitis pigmentosa.

What is retinitis pigmentosa?

Retinitis pigmentosa is a genetic disorder, characterised by progressive loss of sight. In retinitis pigmentosa, some cells in the retina (the light-sensitive part of the eye), called rods and cones, are progressively damaged and disappear. These cells are fundamental for eyesight. Retinitis pigmentosa is chronically debilitating due to progressive loss of vision.

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

At the time of designation, retinitis pigmentosa affected less than 3 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than149,000 people, and is below the threshold for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and 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 498,000,000 (Eurostat 2006).

What treatments are available?

At the time of submission of the application for orphan designation, no satisfactory method had been authorised in the European Union for the treatment of the condition. Treatment of patients with retinitis pigmentosa primarily involved genetic counselling, and general support such as information and regular medical follow-up.

How is this medicine expected to work?

Recombinant human rod-derived cone viability factor (rh-RdCVF) is a protein, composed of 109 blocks called amino acids. This protein is normally produced by some cells of the retina, called rods. The product is expected to protect other cells in the retina, called cones, from being damaged. By administering additional quantities of this substance directly inside the eye, it is expected that vision will be preserved for a longer time.

What is the stage of development of this medicine?

The effects of recombinant human rod-derived cone viability factor were evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials in patients with retinitis pigmentosa had been initiated.

The medicinal product was not authorised anywhere in the world for retinitis pigmentosa or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 10 October 2007 a positive opinion recommending the grant of the above-mentioned 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 Community) 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.