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Orphan designation

On 29 October 2007, orphan designation (EU/3/07/489) was granted by the European Commission to Novagali Pharma SA, France, for ciclosporin for treatment of Herpes simplex virus stromal keratitis.

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 Herpes simplex virus stromal keratitis?

Herpes simplex virus stromal keratitis is an inflammatory disease of the cornea (the central transparent tissue in the front of the pupil, in the eye) associated with infection by the Herpes simplex virus. This inflammation leads to dense scarring of the cornea. The condition is chronically debilitating due to possible loss of sight.

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

At the time of designation, Herpes simplex virus stromal keratitis affected less than 1.6 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 80,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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 treatments are available?

Various medicinal products were authorised for the treatment of Herpes simplex virus stromal keratitis in the Community at the time of submission of the application for orphan drug designation.

Ciclosporin might be of potential significant benefit for the treatment of Herpes simplex virus stromal keratitis. Ciclosporin might help to achieve better overall control of the condition. This assumption will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

How is this medicine expected to work?

Ciclosporin is an immunosuppressor, in other words it acts by inhibiting (blocking) the cells from the defence system involved in the inflammation process. The inflammation in stromal keratitis is thought to be the most important cause of the damage caused to the corneal structure by the viral infection.

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 Herpes simplex virus stromal keratitis were initiated.

Ciclosporin was not authorised anywhere in the world for the treatment of Herpes simplex virus stromal keratitis, 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/conditionTreatment of herpes simplex virus stromal keratitis
Date of decision28/10/2007
Orphan decision numberEU/3/07/489

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.