EU/3/09/707

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Orphan designation

On 28 January 2010, orphan designation (EU/3/09/707) was granted by the European Commission to Actelion Registration Limited, United Kingdom, for macitentan for the treatment of idiopathic pulmonary fibrosis.

What is idiopathic pulmonary fibrosis?

Idiopathic pulmonary fibrosis is a long-term disease of the lungs characterised by the progressive formation of hard tissue in the lining (endothelium) of the lungs. ‘Idiopathic’ means that the cause of the disease is unknown. As the tissue becomes thicker and forms scars, the lungs become unable to work normally, reducing the transfer of oxygen from the air into the blood. Patients with idiopathic pulmonary fibrosis have a persistent cough, frequent lung infections and severe shortness of breath that worsens over time.

Idiopathic pulmonary fibrosis is a long-term debilitating disease that may be life-threatening because of problems with breathing.

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

At the time of designation, idiopathic pulmonary fibrosis affected approximately 2.7 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 136,000 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).

What treatments are available?

At the time of application, there were no authorised medicines for the treatment of idiopathic pulmonary fibrosis. Only medicines to relieve the symptoms of the disease were available, 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?

Macitentan is expected to work as an ‘endothelin receptor antagonist’. This means that it is expected to block 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. It has a wide range of effects, including causing fibrosis (scar tissue), cell proliferation and inflammation. By blocking the endothelin receptor, macitentan is expected to block the activity of endothelin-1, reducing the symptoms of idiopathic pulmonary fibrosis.

What is the stage of development of this medicine?

The effects of macitentan have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with the designated product in patients with idiopathic pulmonary fibrosis were ongoing.
At the time of submission, macitentan was not authorised anywhere in the EU for idiopathic pulmonary fibrosis. Orphan designation of macitentan had been granted in the United States of America for idiopathic pulmonary fibrosis.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 5 November 2009 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 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>Macitentan</p>
Active substanceMacitentan
Medicine Name
Disease/conditionTreatment of idiopathic pulmonary fibrosis
Date of decision28/01/2010
OutcomePositive
Orphan decision numberEU/3/09/707

Review of designation

Contact point:

Sponsor’s contact details:

Actelion Registration Limited
BSI Building 13th floor
389 Chiswick High Road
London W4 4AL
United Kingdom
Telephone: + 44 208 987 3320
Telefax: + 44 208 987 3322
E-mail: registration@actelion.com

Patient associations’ contact points:

British Lung Foundation (Helpline)
73 – 75 Goswell Road
London EC1V 7ER
United Kingdom
Telephone: +44 8458 50 50 20
Telefax: +44 151 224 7779
E-mail: enquiries@blf-uk.org

Associazione Italiana Malattie Interstiziali o rare del Polmone
Via Etruria 14
00183 Roma
Italy
Telephone: +39 338 782 29 86
Telefax: +39 06 62 20 13 92
E-mail: info@aimip.org

Asociación de Familiares y Enfermos de Fibrosis Pulmonar Idiopática
Lugar de Cobas, 3 - Os Tilos
15894 Teo (A Coruña)
Spain
Telephone: +34 662 536 168
E-mail: afefpi@gmail.com