Overview

On 8 October 2009, orphan designation (EU/3/09/666) was granted by the European Commission to S.L.A. Pharma (UK) Ltd, United Kingdom, for eicosapentaenoic acid for the treatment of familial adenomatous polyposis.

The sponsor’s address was updated in March 2021.

The sponsorship was transferred to SLA Pharma (IRE) Limited, Ireland, in April 2021.

Familial adenomatous polyposis (FAP) is a hereditary disease characterised by the formation of numerous polyps (growths), mainly in the large intestine. Polyps usually start to develop in late childhood and their number varies from hundreds to thousands. Patients with FAP may have blood in the stools, diarrhoea or constipation, abdominal pain (stomach ache) and weight loss for no obvious reason.

FAP is a long-term debilitating disease that may be life threatening because of the high risk of developing into cancer of the large intestine if it is not treated.

At the time of designation, FAP affected less than 1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 50,000 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 504,800,000 (Eurostat 2009).

At the time of designation, celecoxib was authorised in the EU for the treatment of FAP in addition to surgery (to remove the polyps) and endoscopic monitoring (to check if polyps are developing, using an endoscope, a thin tube that allows a doctor to look inside the gut).

The sponsor has provided sufficient information to show that eicosapentaenoic acid might be of significant benefit for patients with FAP because early studies indicate that it might be effective in reducing the number of polyps and might have fewer side effects than existing treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

Eicosapentaenoic acid is an omega-3 fatty acid that is obtained from fish oil. The way that eicosapentaenoic acid works in FAP is not fully understood, but increasing the intake of eicosapentaenoic acid has been shown to change the composition of fats in the cell membrane. In particular, it is thought that eicosapentaenoic acid replaces another fat in the cell membrane called arachidonic acid, which is involved in the inflammation process. Replacing arachidonic acid with eicosapentaenoic acid is expected to reduce inflammation. This may help to suppress the formation of polyps in the intestine.

The effects of eicosapentaenoic acid have been evaluated in experimental models.

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

At the time of submission, eicosapentaenoic acid was not authorised anywhere in the EU for FAP or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 8 July 2009 recommending the granting of this designation.

  • 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.

EU/3/09/666: Public summary of positive opinion for orphan designation of eicosapentaenoic acid for the treatment of familial adenomatous polyposis

Key facts

Active substance
Eicosapentaenoic acid
Intended use
Treatment of familial adenomatous polyposis (FAP)
Orphan designation status
Positive
EU designation number
EU/3/09/666
Date of designation
Sponsor

SLA Pharma (IRE) Limited
Unit 3d North Point House
North Point Business Park
New Mallow Road
Cork
Ireland
Email: admin@slapharma.com

EMA list of opinions on orphan medicinal product designation

EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Patients' organisations

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

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

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