EU/3/07/455

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Orphan designation

On 22 October 2007, orphan designation (EU/3/07/455) was granted by the European Commission to Novagali Pharma SA, France, for ciclosporin for the prevention of corneal graft rejection.

What is corneal graft rejection?

After transplantation of the cornea (the central transparent part of the eye, external to the iris), there is a risk that the transplant (graft) will not be accepted by the patient’s immune system, which would lead to a phenomenon called “rejection”.
The cornea does not have blood vessels, so it is usually difficult for white blood cells and antibodies (both agents of the immune system and involved in rejection) to reach it and activate rejection mechanisms. For this reason, rejection tends to occur less frequently in corneal transplants compared to other organs that do have blood vessels, such as the kidney. However, it is still a feared complication as it can lead to loss of vision in the affected eye.

What are the methods of prevention available?

Currently, no satisfactory methods exist in the European Union that were authorised at the time of application, for the prevention of corneal graft rejection. However, eye drops containing corticosteroids (that reduce inflammation and suppress the immune system) are commonly prescribed after corneal transplantation to prevent rejection; other drugs that have been employed (although they are not authorized in the European Union) are ciclosporin and tacrolimus, both for the treatment and the prevention of corneal graft rejection.

What is the estimated number of patients at risk of developing the condition*?

Based on the information provided by the sponsor and previous knowledge of the Committee, the population of patients eligible for prevention of corneal graft rejection was considered to correspond to approximately 1.1 in 10,000 persons in the European Union, which, at the time of designation, corresponded to about 55,000 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?

Ciclosporin is an immunosuppressor, in other words it acts by reducing the activity of the immune system. Ciclosporin given by mouth or as an injection is authorized for the prevention and treatment of the rejection of many other organs, including the kidney, the heart and the liver. Local administration (as eye drops) of ciclosporin is expected to reduce the chance of corneal graft rejection.

What is the stage of development of this medicinal product?

The effects of ciclosporin were evaluated in experimental models. At the time of submission of the application for orphan designation, no clinical trials in patients with corneal transplants were initiated.

Ciclosporin was not authorised anywhere in the world for the prevention of corneal graft rejection, 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 12 September 2007 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>Ciclosporin</p>
Active substanceCiclosporin
Medicine Name
Disease/conditionPrevention of corneal graft rejection
Date of decision22/10/2007
OutcomePositive
Orphan decision numberEU/3/07/455

Review of designation

Sponsor’s contact details:

Novagali Pharma SA
Bât Genavenir IV-1 rue Pierre Fontaine
91 058-Evry Cedex
France
Telephone: +33 1 69 87 40 23
Telefax: +33 1 69 87 40 33
E-Mail: contact@novagali.com

Patients’ associations contact points:

Keratos - European association on ocular surface diseases and lachrymal dysfunctions
55 avenue de la République 
93170 Bagnolet 
France
Telephone: +33 9 54 09 76 88
E-mail: keratos_contact@hotmail.com

Eyecare Trust
PO Box 804
Aylesbury
HP20 9DF Bucks
United Kingdom
Telephone: +44 845 129 5001
Telefax: +44 845 129 5001
E-mail: info@eyecaretrust.org.uk

Action for Blind People
14-16 Verney Road 
SE16 3DZ  London 
United Kingdom 
Freephone: 0800 915 4666 (for UK residents) 
Telephone: +44 20 7635 4800