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Orphan designation

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in April 2014 on request of the Sponsor.

The sponsorship was transferred to Actelion Registration Limited, United Kingdom in July 2001.

On 17 March 2003, orphan designation (EU/3/03/139) was granted by the European Commission to Actelion Registration Limited, United Kingdom, for bosentan for the treatment of systemic sclerosis.

Bosentan for treatment of systemic sclerosis has been authorised in the EU as Tracleer since 7 June 2007.

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

What is systemic sclerosis?

Systemic sclerosis involves the abnormal growth of connective tissue, which supports the skin and internal organs. The abnormal growth is due to too much production of a protein called collagen. The disease not only includes the skin, but also involves the tissues around the blood vessels and major organs. Systemic sclerosis is typically broken down into diffuse and limited disease. In diffuse form, the skin becomes thicker over much of the body, such as the upper arms, upper legs, chest, and stomach. Inside the body, the disease can damage key organs such as the heart, lungs, and kidneys. The limited form typically affects the skin only in certain areas: the fingers, hands, face, lower arms, and legs. Many people with limited disease have Raynaud's phenomenon (a condition in which the small blood vessels of the hands and/or feet contract in response to cold or anxiety: they turn white and cold, then blue) for years before the skin starts to become thicker. People with limited disease are more prone to develop hypertension of the heart-lung circulation (pulmonary hypertension) than people with diffuse disease. Due to the thicker skin and the damage to key internal organs, the disease is considered chronically debilitating.

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

At the time of designation, systemic sclerosis affected between 0.3 and 1.3 in 10,000 people in the European Union (EU). This was equivalent to a total of between 11,000 and 50,000 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. At the time of designation, this represented a population of 382,800,000 (Eurostat 2003).

What treatments are available?

There is no treatment that stops the build-up of collagen. Available treatment is aimed at relieving symptoms and limiting damage. Several products with anti-inflammatory activity (products that limit the reaction of tissues against damage) were authorised for the condition in some countries in the Community at the time of submission of the application for orphan drug designation. Bosentan might be of potential significant benefit for the treatment of systemic sclerosis in particular with regard to efficacy in specific disease related complications. This benefit will have to be confirmed at the time of marketing authorisation and will be necessary to maintain the orphan status.

How is this medicine expected to work?

Bosentan opposes the effect of a substance called endothelin-1. This substance can cause narrowing of blood vessels and may also play an important role in the disease process of systemic sclerosis. Therefore, bosentan could play an important role in treating major complications of the disease.

What is the stage of development of this medicine?

Bosentan has previously received orphan drug designation and has been authorised in the European Union to treat (pulmonary hypertension), which included patients with systemic sclerosis. 

At the time of submission of the application for the current orphan designation, further clinical trials in patients with systemic sclerosis were ongoing.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 7 February 2003 recommending the granting of this designation.

Update: Bosentan (Tracleer) has been authorised in the EU since 22 March 2007 for the treatment of new digital ulcers in patients with systemic sclerosis and active digital ulcers.

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/3/03/139: Public summary of positive opinion for orphan designation of bosentan for treatment of systemic sclerosis (English only) 2007-05-30 2014-05-08

Key facts

Product details for <p>Bosentan</p>
Active substanceBosentan
Medicine NameTracleer
Disease/conditionTreatment of systemic sclerosis (scleroderma)
Date of decision17/03/2003
Orphan decision numberEU/3/03/139

Review of designation

Related information:

Sponsor's contact details:

Actelion Registration Limited
Chiswick Tower 13th floor
389 Chiswick High Road
London W4 4AL
United Kingdom
Tel. +44 (0)20 8987 3320
Fax +44 (0)20 8987 3322

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.