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Orphan designation

On 5 August 2013, orphan designation (EU/3/13/1180) was granted by the European Commission to Celgene Europe Limited, United Kingdom, for apremilast for the treatment of Behçet’s disease.

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in April 2017 on request of the Sponsor.

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 and blindness.

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 was equivalent to a total of fewer than 51,000 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, treatment for Behçet’s disease 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 also approved for Behçet’s disease in some EU Member States.

The sponsor has provided sufficient information to show that apremilast might be of significant benefit for patients with Behçet’s disease because early studies in patients showed that the medicine was effective at reducing the number of mouth sores. This assumption 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?

Apremilast is expected to work by blocking the production of various messenger molecules, including interleukins and tumour necrosis factors. These messengers are involved in causing inflammation and are found at high levels in patients with Behçet’s disease. By blocking their production, apremilast reduces the symptoms of Behçet’s disease, such as the sores in the mouth.

What is the stage of development of this medicine?

The effects of apremilast have been evaluated in experimental models.

At the time of submission of the application for orphan designation, one clinical trial with apremilast in patients with Behçet’s disease had been conducted.

At the time of submission, apremilast was not authorised anywhere in the EU for Behçet’s disease. Orphan designation of apremilast had been granted in the United States for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 11 July 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.

Name Language First published Last updated
EU/3/13/1180: Public summary of opinion on orphan designation: Apremilast for the treatment of Behçet’s disease (English only) 2013-09-13 2017-05-17

Key facts

Product details for <p>Apremilast</p>
Active substanceApremilast
Medicine Name
Disease/conditionTreatment of Behçet’s disease
Date of decision05/08/2013
Orphan decision numberEU/3/13/1180

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

Celgene Europe Limited
1 Longwalk Road
Stockley Park
UB11 1DB
United Kingdom
Tel. +44 (0)20 8831 8300
Fax +44 (0)20 8831 8301

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.