• Email
  • Help

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.

In March 2015, Novagali Pharma SA changed name to SANTEN SAS.

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

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

At the time of designation, the number of patients at risk of corneal graft rejection was estimated to be approximately 1.1 people in 10,000 in the European Union (EU). This was equivalent to a total of around 55,000 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).

*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.
At the time of designation, this represented a population of 500,300,000 (Eurostat 2007).

What methods of prevention are 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.

How is this medicine expected to work?

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 medicine?

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.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 12 September 2007 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.

Key facts

Product details for <p>Ciclosporin</p>
Active substanceCiclosporin
Medicine Name
Disease/conditionPrevention of corneal graft rejection
Date of decision22/10/2007
Orphan decision numberEU/3/07/455

Review of designation

Sponsor’s contact details:

Bâtiment Genavenir IV
1, Rue Pierre Fontaine
F-91058-Evry cedex
Tel. +33 1 69 87 40 20
Fax +33 1 69 87 40 30

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.