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Orphan designation

On 5 September 2008, orphan designation (EU/3/08/559) was granted by the European Commission to Actelion Registration Limited, United Kingdom, for bosentan for the treatment of idiopathic pulmonary fibrosis.

What is idiopathic pulmonary fibrosis?

Fibrosis is the formation of scar tissue that is part of the natural repair process of the body following tissue damage. Idiopathic pulmonary fibrosis consists of a chronic inflammation and progressive formation of fibrous tissue in the lungs. ‘Idiopathic’ means that the cause of the disease is unknown. The progressive formation of scars makes the lungs unable to work normally, reducing the transfer of oxygen from the air into the blood. Patients with idiopathic pulmonary fibrosis have a persistent cough, severe shortness of breath that gets worse and frequent lung infections.

Idiopathic pulmonary fibrosis is a long-term, debilitating and life-threatening disease.

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

At the time of designation idiopathic pulmonary fibrosis affected approximately 1.9 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 95,000 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 502,282,000 (Eurostat 2008).

What treatments are available?

No satisfactory methods were authorised for the treatment of idiopathic pulmonary fibrosis at the time of application. Only medicines to relieve the symptoms of the disease were used, including corticosteroids (anti-inflammatory medicines) and medicines that reduce the activity of the immune system (the body’s natural defences). In some patients, the disease may need to be treated with a lung transplant.

How is this medicine expected to work?

Bosentan is an ‘endothelin receptor antagonist’. This means that it blocks the receptor to which a substance called ‘endothelin-1’ normally attaches itself. Endothelin-1 is a naturally occurring substance that is released from the endothelium (the cells lining the blood vessels). It has a wide range of effects, including causing fibrosis, cell proliferation and inflammation. By blocking the endothelin receptor, bosentan blocks the activity of endothelin-1, reducing the symptoms of idiopathic pulmonary fibrosis.

What is the stage of development of this medicine?

The effects of bosentan were evaluated in experimental models.

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

At the time of submission, bosentan was not authorised anywhere in the world for the treatment of idiopathic pulmonary fibrosis or designated as an orphan medicinal product elsewhere for this condition. The product was authorised in the Community for the treatment of pulmonary arterial hypertension and the treatment of digital ulcers in patients with systemic sclerosis.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted a positive opinion on 11 June 2008 recommending the granting of the above-mentioned 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>Bosentan</p>
Active substanceBosentan
Medicine Name
Disease/conditionTreatment of idiopathic pulmonary fibrosis
Date of decision05/09/2008
Orphan decision numberEU/3/08/559

Review of designation

Sponsor’s contact details:

Actelion Registration Limited
BSI Building 13th floor
389 Chiswick High Road
London W44 4AL
United Kingdom
Telephone: +41 61 565 66 58
Telefax: +41 61 565 64 10

Patients’ associations contact points:

British Lung Foundation (Helpline)
73 – 75 Goswell Road
London EC1V 7ER
United Kingdom
Telephone: +44 8458 50 50 20 (Mon to Fri 10:00 to 18:00)
Telefax: +44 151 224 7779

Belangenvereniging Longfibrosepatiënten Nederland
Postbus 5800 6202 AZ
Telephone: +31 43 361 9266

Associazione Italiana Malattie Interstiziali o rare del Polmone
Via Etruria 14 00183
Telephone: +39 338 782 29 86
Telefax: +39 06 62 20 13 92