EU/3/06/412

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Orphan designation

On 3 November 2006, orphan designation (EU/3/06/412) was granted by the European Commission to Jose Manuel Cela Lopez, Spain, for 4,7,10,13,16,19-docosahexaenoic acid for the treatment of retinitis pigmentosa.

The sponsorship was transferred to Celavista Pharmaceuticals Limited, Ireland, in August 2011 and subsequently to Natac Pharma SL, Spain, in May 2013.

For a list of the administrative updates to this public summary of opinion, please refer to the PDF document below.

What is retinitis pigmentosa?

Retinitis pigmentosa is a genetic (hereditary) 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 eventually 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 3.5 in 10,000 people in the European Union (EU)*. This is equivalent to a total of less than 175,000 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 459,700,000 (Eurostat 2004).

What treatments are available?

At the time of submission of the application for orphan designation, no satisfactory methods 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?

4,7,10,13,16,19-Docosahexaenoic acid (DHA) is a so-called omega-three fatty acid. These acids are an integral part of the outer lipid cell membrane of all the cells in the body (the thin double layer of fatty acids that surrounds and protects cells). In the cell membrane of the rods and cones of the eye, DHA is even more abundant than in the rest of the body. It is important for the light absorption activity of these cells. There is some evidence that suggest that patients affected by retinitis pigmentosa have decreased levels of DHA. According to the sponsor, 4,7,10,13,16,19-docosahexaenoic acid will thus reduce the effects of the disease.

What is the stage of development of this medicine?

The effects of 4,7,10,13,16,19-docosahexaenoic acid have been evaluated in experimental models.

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

4,7,10,13,16,19-Docosahexaenoic acid was not authorised anywhere worldwide for the treatment of retinitis pigmentosa, nor designated as orphan medicinal product elsewhere for this condition, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 4 October 2006 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 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.

Key facts

Product details for <p>4,7,10,13,16,19-docosahexaenoic acid</p>
Active substance4,7,10,13,16,19-docosahexaenoic acid
Medicine Name
Disease/conditionTreatment of retinitis pigmentosa
Date of decision03/11/2006
OutcomePositive
Orphan decision numberEU/3/06/412

Review of designation

Sponsor’s contact details:

Natac Pharma S.L.
C/ Faraday 7
28049 Madrid
Spain
Tel. +34 91 827 64 70
Fax +34 91 827 66 99

Patients’ associations contact points:

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.