EU/3/05/346

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Orphan designation

On 24 January 2006, orphan designation (EU/3/05/346) was granted by the European Commission to Biotech Tools SA, Belgium, for E. Coli heat-shock protein 70 with bovine retinal S-antigen for the treatment of autoimmune uveitis.

What is autoimmune uveitis?

The term uveitis covers clinical symptoms of inflammation of the uvea, the middle layer of the eye - between the sclera and retina. Autoimmune uveitis is a type of uveitis where a reaction to an individual’s own tissues (in this case in the eye) occurs because the body’s defence system mistakenly recognises it as foreign tissue and tries to destroy it. This may occur in association with other autoimmune illnesses that include, amongst others, various types of arthritis and some bowel diseases. In many cases the cause of the uveitis cannot be found.
Uveitis can affect one eye or both eyes and may cause varying degrees of discomfort or pain, with or without blurring of vision. The eye may be red or appear white. Autoimmune uveitis is chronically debilitating as it may lead to structural damage to the tissues in the eye, cataracts (opacity of the transparent structures of the eyes impairing vision or causing blindness) and sight loss.

What are the methods of treatment available?

Various medicinal products were available for the treatment of autoimmune uveitis and were authorised in some Member States in the Community at the time of submission of the application for orphan drug designation.
E. Coli heat-shock protein 70 with bovine retinal S-antigen might be of potential significant benefit for the treatment of autoimmune uveitis. This medicinal product might help to achieve better overall control of the condition. The assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

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

According to the information provided by the sponsor, autoimmune uveitis was considered to affect about 207,000 persons in the European Union.

*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?

E. Coli heat-shock protein 70 with bovine retinal S-antigen mimicks a portion of one of the eye natural proteins against which the patient’s immune system reacts thus resulting in autoimmune uveitis. Once administered to the patient, E. Coli heat-shock protein 70 with bovine retinal S-antigen is expected to induce tolerance of the body’s defence system to the natural proteins of the eye that are mistakenly recognised as foreign tissues.

What is the stage of development of this medicinal product?

The evaluation of the effects of E. Coli heat-shock protein 70 with bovine retinal S-antigen in experimental models is ongoing.
At the time of submission of the application for orphan designation, no clinical trials in patients with autoimmune uveitis were initiated.
E. Coli heat-shock protein 70 with bovine retinal S-antigen was not authorised anywhere worldwide for autoimmune uveitis or designated as orphan medicinal product elsewhere for this condition, 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 December 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.

Key facts

Product details for <p>E. Coli heat-shock protein 70 with bovine retinal S-antigen</p>
Active substanceE. Coli heat-shock protein 70 with bovine retinal S-antigen
Medicine Name
Disease/conditionTreatment of autoimmune uveitis
Date of decision24/01/2006
OutcomePositive
Orphan decision numberEU/3/05/346

Review of designation

Sponsor’s contact details:

Biotech Tools SA
Rue de Ransbeek, 230 A
1120 Brussels
Belgium
Telephone: +32 2 26 40 39 0
Telefax: +32 2 26 40 39 9
E-mail: thierry.legon@biotech.be

Patients’ associations contact points:

INFLAM’OEIL
France
No postal address available
Telephone: +33 6 12 83 93 63
E-mail: inflamoeil@yahoo.fr

Uveitis Information Group (UIG)
South House
Sweening
Vidlin
Shetland Isles
ZE2 9QE
United Kingdom
Telephone: +44 18 06 57 73 10
E-mail: info@uveitis.net

Geschäftsstelle DUAG e.V.
Forsthausstrasse 6
35792 Löhnberg
Germany
Telephone: + 49 64 71 98 05 7
Telefax: +49 64 71 98 05 8
E-Mail: duag-ev@web.de