EU/3/08/533

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Orphan designation

On 1 April 2008, orphan designation (EU/3/08/533) was granted by the European Commission to Genzyme BV, The Netherlands, for recombinant human monoclonal antibody against transforming growth factor beta-1, 2 and 3 for the treatment of idiopathic pulmonary fibrosis.

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 is thought to consist of a chronic inflammation (a response of the body to the injury caused to the tissue) and progressive formation of fibrous tissue in the walls of the small chambers 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 respiratory symptom and 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 were used (such as corticosteroids and medicinal products that suppress the immune system) at the time of the submission of the application. In some patients, lung transplantation was performed.

What is the estimated number of patients affected by idiopathic lung fibrosis*?

Based on the information provided by the sponsor and previous knowledge of the Committee, idiopathic lung fibrosis was considered to affect approximately 2.7 in 10,000 persons in the European Union, which, at the time of designation, corresponded to about 134,000 persons.

* 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 27), Norway, Iceland and Lichtenstein. This represents a population of 498,000,000 (Eurostat 2006). 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?

Transforming growth factor beta (TGF beta) is a protein that can develop multiple functions. This protein comes in three forms (TGF beta-1, 2 and 3). TGF beta plays a specific role in accumulation of connective tissue that is important for scarring and wound healing. In idiopathic pulmonary fibrosis, these processes are abnormally high and lead to increased fibrosis. Therefore, it is thought that the excess fibrosis can be modified by blocking TGF beta.

Antibodies are proteins in the body that can identify and neutralize specific targets, and they are normally used by the immune system. Recombinant human monoclonal antibody against transforming growth factor beta-1, 2 and 3 is an antibody artificially produced that is able to recognise and block all three forms of TGFβ.

What is the stage of development of this medicinal product?

The effects of recombinant human monoclonal antibody against transforming growth factor beta-1, 2 and 3 were evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials in patients with idiopathic lung fibrosis were ongoing.

Human monoclonal antibody against transforming growth factor beta-1, 2 and 3 was not authorised anywhere worldwide for idiopathic pulmonary fibrosis, at the time of submission.

Orphan designation of human monoclonal antibody against transforming growth factor beta-1, 2 and 3 was granted in the United States for idiopathic pulmonary fibrosis.

According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 6 February 2008 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.

Key facts

Product details for <p>Recombinant human monoclonal antibody against transforming growth factor beta-1, 2 and 3</p>
Active substanceRecombinant human monoclonal antibody against transforming growth factor beta-1, 2 and 3
Medicine Name
Disease/conditionTreatment of idiopathic pulmonary fibrosis
Date of decision01/04/2008
OutcomePositive
Orphan decision numberEU/3/08/533

Review of designation

Sponsor’s contact details:

Genzyme Europe BV
Gooimeer 10
1411 DD Naarden
The Netherlands
Telephone: +31 035 699 1452
Telefax: +31 035 699 1444
E-mail: Miriam.titulaer@genzyme.com

Patients’ associations contact points:

Associazione Italiana Malattie Interstiziali o rare del Polmone
Via Etruria 14
00183 Roma
Italy
Telephone: +39 338 7822986

Contact a Family
209-211 City Road 
London EC1V 1JN
United Kingdom
Telephone: +44 20 76 08 87 00
Helpline: 0808 808 3555 (for UK residents only)
Telefax: +44 20 76 08 87 01
Textphone 0808 808 3556 (for UK residents only)
E-mail: info@cafamily.org.uk