EU/3/04/219 - orphan designation for treatment of autoimmune uveitis

HLA-B27-derived peptide (amino acid 125-138)

Overview

On 2 September 2004, orphan designation (EU/3/04/219) was granted by the European Commission to Lynkeus BioTech GmbH, Germany, for HLA-B27-derived peptide (amino acid 125-138) for the treatment of autoimmune uveitis.

The sponsorship was transferred to Dr Gerhild Wildner, Germany, in August 2005.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

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 is never 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 is the estimated number of patients affected by the condition?

At the time of designation, autoimmune uveitis affected approximately 4.5 in 10,000 people in the European Union (EU)*. This is equivalent to a total of around 209,000 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 25), Norway, Iceland and Liechtenstein.
At the time of designation, this represented a population of 464,200,000 (Eurostat 2004).

What treatments are 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.

HLA-B27-derived peptide (amino acid 125-138) 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.

This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

How is this medicine expected to work?

HLA-B27-derived peptide (amino acid 125-138) 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, HLA-B27-derived peptide 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 medicine?

The evaluation of the effects of HLA-B27-derived peptide (amino acid 125-138) in experimental models is ongoing. At the time of submission of the application for orphan designation, clinical trials in patients with autoimmune uveitis were ongoing.

HLA-B27-derived peptide (amino acid 125-138) was not marketed anywhere worldwide for autoimmune uveitis or designated as orphan medicinal product elsewhere for this condition, at the time of submission.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 22 July 2004 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 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/3/04/219: Public summary of positive opinion for orphan designation of HLA-B27-derived peptide (amino acid 125-138) for the treatment of autoimmune uveitis

Adopted Reference Number: EMEA/COMP/391/2004 Rev.3

Key facts

• Active substance:
  HLA-B27-derived peptide (amino acid 125-138)
• Intended use:
  Treatment of autoimmune uveitis
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/04/219
• Date of designation:
  02/09/2004
• Sponsor:

  Dr Gerhild Wildner ; Clinic of the University of Munich ; Dept. of Opthalmology; Mathildenstr. 8; 80336 Munich; Germany; Tel. [phone]; Fax [phone]; E-mail: [e-mail]