EU/3/10/796

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Orphan designation

On 1 October 2010, orphan designation (EU/3/10/796) was granted by the European Commission to XOMA Ireland Ltd, Ireland, for recombinant humanised anti-human interleukin-1 beta monoclonal antibody for the treatment of Behçet’s disease.

The sponsorship was transferred to Les Laboratoires Servier, France, in December 2011.

What is Behçet’s disease?

Behçet’s disease is an auto-immune disease in which the immune system attacks its own blood vessels. The exact cause of the disease is unknown. As a result of the damage to the blood vessels, patients develop symptoms such as painful sores in the mouth and on the genitals, inflammation inside the eye and skin problems. The inflammation inside the eye can lead to blurred vision, pain, and redness.

Behçet’s disease is a long-term debilitating disease because the symptoms such as the sores in the mouth and on the genitals and inflammation of the blood vessels throughout the body can last several weeks and cause permanent tissue damage.

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

At the time of designation, Behçet’s disease affected less than 1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 51,000 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 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 506,500,000 (Eurostat 2010).

What treatments are available?

At the time of designation, there were no treatments that could cure Behçet’s disease. Treatment focussed on relieving the symptoms of the disease, reducing discomfort and preventing serious complications. Commonly used treatments included medicines that reduce inflammation, such as steroids. Colchicine was approved for Behçet’s disease in one Member State.

The sponsor has provided sufficient information to show that recombinant humanised anti-human interleukin-1 beta monoclonal antibody might be of significant benefit for patients with Behçet’s disease because it works in a different way to existing treatments, and because early studies indicate that it might improve the ocular (eye) symptoms of the disease. These assumptions will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

This medicine is a monoclonal antibody (a type of protein) that has been designed to recognise and attach to a chemical messenger in the blood called interleukin-1 beta. Interleukin-1 beta is involved in the process of inflammation and is thought to play a role in Behçet’s disease development. By attaching to interleukin-1 beta, this medicine is expected to block its activity, helping to relieve the symptoms of the disease.

What is the stage of development of this medicine?

The effects of recombinant humanised anti-human interleukin-1 beta monoclonal antibody have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with this medicine in patients with Behçet’s disease were ongoing.

At the time of submission, this medicine was not authorised anywhere in the EU for Behçet’s disease 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 2010 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>Recombinant humanised anti-human interleukin-1 beta monoclonal antibody</p>
Active substanceRecombinant humanised anti-human interleukin-1 beta monoclonal antibody
Medicine Name
Disease/conditionTreatment of Behçet’s disease
Date of decision01/10/2010
OutcomePositive
Orphan decision numberEU/3/10/796

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:

Les Laboratoires Servier
50, rue Carnot, 92284
Suresnes, Cedex, France
Telephone: +33 1 55 72 34 93
Telefax: +33 1 55 72 54 12
vanessa.daros@fr.netgrs.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.