EU/3/09/724

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Orphan designation

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.

What is chronic non-infectious uveitis?

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.

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

At the time of designation, chronic non-infectious uveitis affected less than 4.8 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 242,000 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: 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. This represents a population of 504,800,000 (Eurostat 2009).

What treatments are available?

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.

How is this medicine expected to work?

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.

What is the stage of development of this medicine?

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.

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 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.

Key facts

Product details for <p>Recombinant human monoclonal antibody to human interleukin (IL)-17A of the IgG1/k class</p>
Active substanceRecombinant human monoclonal antibody to human interleukin (IL)-17A of the IgG1/k class
Medicine Name
Disease/conditionTreatment of chronic non-infectious uveitis
Date of decision02/02/2010
OutcomePositive
Orphan decision numberEU/3/09/724

Review of designation

Sponsor’s contact details:

Novartis Europharm Limited
Wimblehurst Road
Horsham
West Sussex RH12 5AB
United Kingdom
Telephone: +44 1403 27 28 27
Telefax: +44 1403 32 30 60
E-mail: gesa.pellier@novartis.com

Patient associations’ contact points:

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

INFLAM’OEIL
France
Telephone: +33 6 12 83 93 63
E-mail: inflamoeil@yahoo.fr

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