EU/3/05/281 - orphan designation for treatment of idiopathic pulmonary fibrosis

Interferon gamma

Overview

Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in May 2007 on request of the sponsor.

On 27 May 2005, orphan designation (EU/3/05/281) was granted by the European Commission to Mondobiotech Laboratories Anstalt, Liechtenstein, for interferon gamma for the treatment of idiopathic pulmonary fibrosis.

The sponsorship was transferred to InterMune Europe Ltd, United Kingdom, in September 2006.

What is idiopathic pulmonary fibrosis?

Fibrosis is the formation of scar tissue as part of the natural repair process of the body following tissue damage. Idiopathic pulmonary fibrosis consists of a chronic inflammation (a response to the injury caused to the tissue) and progressive formation of fibrous tissue in the walls of the small cells containing air in the lungs. Since the injury causing these changes is unknown, it is called idiopathic. The progressive formation of scars impairs the normal functions of lung tissue, which are to enable exchange of oxygen and carbon dioxide between air and blood. The symptoms developed are persistent cough, progressive severe shortness of breath and recurrent lung infections.

Idiopathic pulmonary fibrosis is a chronically debilitating and life threatening disease due to the progression of symptoms, severe respiratory complications and short life expectancy.

What are the methods of treatment available?

No satisfactory methods exist that were authorised at the time of application. Only symptomatic treatments to reduce the inflammation were used (corticosteroids and medicinal products that suppress the immune system) or for some patients lung transplantation was performed.

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

According to the information provided by the sponsor, idiopathic pulmonary fibrosis was considered to affect about 138,000 persons in the European Union.

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*Disclaimer: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed based on data from the European Union (EU 25), Norway, Iceland and Lichtenstein. This represents a population of 459,700,000 (Eurostat 2004). This estimate is based on available information and calculations presented by the sponsor at the time of the application.

How is this medicinal product expected to act?

Scar formation (fibrosis) is regulated by several substances produced in the body during the inflammation that precedes fibrosis. These substances stimulate the so-called fibroblasts (the main cells responsible for formation of the fibrosis). Although it is not yet fully understood how interferon gamma acts in idiopathic pulmonary fibrosis, it could stimulate the production of proteins that would prevent fibrosis and re-establish the balance between the molecules that stimulate fibrosis and those that prevent it happening.

What is the stage of development of this medicinal product?

The effects of interferon gamma 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.

Interferon gamma was not marketed anywhere worldwide for idiopathic pulmonary fibrosis, at the time of submission.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 7 April 2005 a positive opinion recommending the grant of the above-mentioned designation.

Opinions on orphan medicinal products designations are based on the following cumulative criteria:

• the seriousness of the condition,
• the existence or not of alternative methods of diagnosis, prevention or treatment and
• either the rarity of the condition (considered to affect not more than five in ten thousand persons in the Community) or the insufficient return of development investments.

Designated orphan medicinal products are still investigational products which were considered for designation on the basis of potential activity. An orphan designation is not a marketing authorisation. As a consequence, demonstration of the quality, safety and efficacy will be necessary before this product can be granted a marketing authorisation.

EU/3/05/281: Public summary of positive opinion of interferon gamma for the treatment of idiopathic pulmonary fibrosis

AdoptedReference Number: EMEA/COMP/130719/2005 Rev.2

Key facts

• Active substance:
  Interferon gamma
• Intended use:
  Treatment of idiopathic pulmonary fibrosis
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/05/281
• Date of designation:
  27/05/2005
• Sponsor:

  InterMune Europe Ltd; Thames House; Wellington Street; London SE18 6NZ; United Kingdom; Tel. [phone]; Fax [phone]; E-mail: [e-mail]