EU/3/07/489

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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.

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 are the methods of treatment 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.

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

Based on the information provided by the sponsor and previous knowledge of the Committee, Herpes simplex virus stromal keratitis was considered to affect less than 1.6 in 10,000 persons in the European Union, which, at the time of designation, corresponded to about 80,000 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 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 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 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.

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/conditionTreatment of herpes simplex virus stromal keratitis
Date of decision28/10/2007
OutcomePositive
Orphan decision numberEU/3/07/489

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’ association(s) contact point(s):

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