EU/3/09/724 - orphan designation for treatment of chronic non-infectious uveitis

recombinant human monoclonal antibody to human interleukin (IL)-17A of the IgG1/k class
OrphanHuman

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in January 2015 on request of the sponsor.

On 2 February 2010, orphan designation (EU/3/09/724) was granted by the European Commission to Novartis Europharm Limited, United Kingdom, for recombinant human monoclonal antibody to human interleukin (IL)-17A of the IgG1/k class for the treatment of chronic non-infectious uveitis.

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

Uveitis is inflammation of the uvea, the middle layer of the eye, which includes the iris. Uveitis can affect one or both eyes and may cause discomfort, pain, and blurring of the vision. In chronic (long-term) non-infectious uveitis the inflammation is usually caused by the body's immune system (the body's natural defences) attacking normal tissue. It is not caused by an infection.

Chronic non-infectious uveitis is a long-term debilitating disease because it may lead to blindness.

At the time of designation, chronic non-infectious uveitis affected less than 4.8 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 243,000 people*, and is below the threshold for orphan designation, which is 5 people in 10,000. This is based on the information provided by the sponsor and knowledge of the Committee for Orphan Medicinal Products (COMP).

*Disclaimer: 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 506,300,000 (Eurostat 2010).

At the time of designation, several medicines were authorised in Member States of the EU for the treatment of chronic non-infectious uveitis. Most treatments aimed to reduce the inflammation by lowering the activity of the immune system.

The sponsor has provided sufficient information to show that recombinant human monoclonal antibody to human interleukin (IL)-17A of the IgG1/k class might be of significant benefit for patients with chronic non-infectious uveitis because early studies indicate that it might improve the treatment of patients with this condition. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

Recombinant human monoclonal antibody to human interleukin (IL)-17A of the IgG1/k class is a monoclonal antibody (a type of protein) that has been designed to recognise and attach to interleukin-17A (IL-17A). IL-17A is a chemical messenger that is thought to be produced in high levels in patients with chronic non-infectious uveitis, causing the inflammation. By attaching to IL-17A, the medicine blocks its activity, helping to relieve the symptoms of the disease.

The effects of recombinant human monoclonal antibody to human interleukin (IL)-17A of the IgG1/k class have been evaluated in experimental models.

At the time of submission of the application for orphan designation, one 16-patient study in patients with chronic non-infectious uveitis had been completed.

At the time of submission, human monoclonal antibody to human interleukin (IL)-17A of the IgG1/k class was not authorised anywhere in the EU for chronic non-infectious uveitis or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 3 December 2009 recommending the granting of this designation.

  • 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 Community) 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/3/09/724: Public summary of opinion on orphan designation of recombinant human monoclonal antibody to human interleukin (IL)-17A of the IgG1/k class for the treatment of chronic non-infectious uveitis

Adopted Reference Number: EMA/COMP/822954/2009 Rev. 2

English (EN) (128.67 KB - PDF)

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Key facts

Active substance
recombinant human monoclonal antibody to human interleukin (IL)-17A of the IgG1/k class
Intended use
Treatment of chronic non-infectious uveitis
Orphan designation status
Withdrawn
EU designation number
EU/3/09/724
Date of designation
Sponsor

Novartis Europharm Limited

EMA list of opinions on orphan medicinal product designation

EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

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