EU/3/07/491 - orphan designation for treatment of idiopathic pulmonary fibrosis

Interferon gamma

Overview

On 29 October 2007, orphan designation (EU/3/07/491) was granted by the European Commission to Foundation for Fatal Rare Diseases, Liechtenstein, for interferon gamma for the treatment of idiopathic pulmonary fibrosis.

The sponsorship was transferred to mondoBIOTECH Laboratories AG, Liechtenstein, in August 2012.

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 chronic inflammation and progressive formation of fibrous tissue 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 the lung tissue, amongst which is oxygenation of the blood. Symptoms of the condition include 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 severe respiratory complications.

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

At the time of designation, idiopathic pulmonary fibrosis affected less than 3 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 150,000 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).

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*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. At the time of designation, this represented a population of 500,300,000 (Eurostat 2007).

What are the methods of treatment available?

No satisfactory methods 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). In some patients, lung transplantation may be performed.

How is this medicine expected to work?

Scar formation (fibrosis) is regulated by several substances produced in the body during the inflammation stage that precedes fibrosis in the repair process. 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.

What is the stage of development of this medicine?

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.

IFN?-1b was authorised in the European Union for the treatment of chronic granulomatous disease, at the time of designation.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 12 September 2007 recommending the granting of this designation.

Opinions on orphan medicinal products designations are based on the following cumulative 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.

EU/3/07/491: Public summary of positive opinion of interferon gamma for the treatment of idiopathic pulmonary fibrosis

Adopted Reference Number: EMEA/COMP/468704/2007 Rev. 1

Key facts

• Active substance:
  Interferon gamma
• Intended use:
  Treatment of idiopathic pulmonary fibrosis
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/07/491
• Date of designation:
  29/10/2007
• Sponsor:

  mondoBIOTECH Laboratories AG; Herrengasse 21; 9490 Vaduz; Liechtenstein; Tel. [phone]; Fax [phone]; E-mail: [e-mail]