EU/3/06/360: Orphan designation for the treatment of vernal keratoconjunctivitis

Vernal keratoconjunctivitis (also known as spring catarrh) is a chronic allergic inflammation (swelling and redness) of the conjunctiva (membrane lining the eyelid and covering the eyeball) and the cornea (clear tissue in the front of the eye that protects deeper structures), most often seen in young children and with seasonal appearance. This inflammation leads to redness, fluid discharge, itching, pain and light sensitivity.

Vernal keratoconjunctivitis is often seen together with other allergic conditions (e.g. eczema and asthma). The condition is chronically debilitating due to possible corneal ulcers and visual loss.

At the time of designation, vernal keratoconjunctivitis affected between 1 to 3 in 10,000 people in the European Union (EU). This was equivalent to a total of around 47,000 to 141,000 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 25), Norway, Iceland and Liechtenstein.
At the time of designation, this represented a population of 468,900,000 (Eurostat 2006).

Some medicinal products were authorised for the treatment of vernal keratoconjunctivitis in the Community at the time of submission of the application for orphan drug designation.
Satisfactory argumentation has been submitted by the sponsor to justify that ciclosporin might be of potential significant benefit for the treatment of vernal keratoconjunctivitis because it may bring better control of the symptoms of the condition. These benefits will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

Ciclosporin is an immunosuppressant drug; it inhibits the function of the cells of the immune system (body's defence to infection and disease). In patients with vernal keratoconjunctivitis it is expected to prevent the inflammation observed in conjuctiva and cornea.

The effects of ciclosporin have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with vernal keratoconjunctivitis were ongoing.

Ciclosporin was not authorised anywhere worldwide for vernal keratoconjunctivitis 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 8 March 2006 recommending the granting of this designation.

• the seriousness of the condition;
• the existence of alternative methods of diagnosis, prevention or treatment;
• and either the rarity of the condition (affecting not more than five in 10,000 people in the Community) or the 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 the quality, safety and efficacy is necessary before a product can be granted a marketing authorisation.