EU/3/06/360 - orphan designation for treatment of vernal keratoconjunctivitis

ciclosporin

Overview

On 6 April 2006, orphan designation (EU/3/06/360) was granted by the European Commission to Novagali Pharma, France, for ciclosporin for the treatment of vernal keratoconjunctivitis.

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

The sponsorship was transferred to Santen Oy, Finland, in January 2016.

Ciclosporin has been authorised in the EU as Verkazia since 6 July 2018.

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

What is 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.

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

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 isbased on the information provided by the sponsor and the knowledge of the Committee for Orphan Medicinal Products (COMP).

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*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).

What treatments are available?

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.

How is this medicine expected to work?

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.

What is the stage of development of this medicine?

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.

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;
• 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.

EU/3/06/360: Public summary of positive opinion for orphan designation of ciclosporin for the treatment of vernal keratoconjunctivitis

AdoptedReference Number: EMEA/COMP/107514/2006 Rev.2

Key facts

• Active substance:
  ciclosporin
• Medicine name:
  Verkazia
• Intended use:
  Treatment of vernal keratoconjunctivitis
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/06/360
• Date of designation:
  06/04/2006
• Sponsor:

  Santen Oy; Niittyhaankatu 20; FI-33720 Tampere; Finland; Tel. [phone]; Fax [phone]; E-mail: [e-mail]

Review of designation

On 31 May 2018 the Committee for Orphan Medicinal Products (COMP) completed its review of the designation EU/3/06/360 for Verkazia (ciclosporin) as an orphan medicinal product in the treatment of vernal keratoconjunctivitis. The COMP assessed whether, at the time of marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other methods of treatment. As other methods of treatment are authorised in the European Union (EU), the COMP also considered whether the medicine is of significant benefit to patients with vernal keratoconjunctivitis. The COMP recommended that the orphan designation of the medicine be maintained¹.

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¹The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with the same therapeutic indication cannot be placed on the market.