EU/3/03/185 - orphan designation for treatment of vernal keratoconjunctivitis

Tacrolimus Hydrate

Overview

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in April 2010 on request of the sponsor.

On 14 January 2004, orphan designation (EU/3/03/185) was granted by the European Commission to Sucampo Pharma Europe Ltd., United Kingdom, for tacrolimus hydrate for the treatment of vernal keratoconjunctivitis.

What is vernal keratoconjunctivitis?

Vernal keratoconjunctivitis (also known as spring catarrh) is a chronic allergic inflammation 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, itch, pain and light sensitivity.

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

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

At the time of designation, vernal keratoconjunctivitis affected less than 1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of fewer than 36,000 people, and is below the threshold 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: The number of patients affected by the condition is estimated and assessed for the purpose of the designation, for a European Community population of 385,000,000 (Eurostat 2002) and may differ from the true number of patients affected by the condition. This estimate is based on available information and calculations presented by the sponsor at the time of the application.

What treatments are available?

Various 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.

Tacrolimus hydrate might be of potential significant benefit for the treatment of vernal keratoconjunctivitis. Tacrolimus hydrate eye drops might help to achieve better overall control 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?

Tacrolimus hydrate is an immunomodulator, in other words it acts by inhibiting the cells from the defence system involved in the inflammation process.

What is the stage of development of this medicine?

The effects of tacrolimus hydrate eye drops were evaluated in experimental models. At the time of submission of the application for orphan designation, clinical trials in patients with vernal keratoconjunctivitis were ongoing.

The medicinal product was not marketed 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 5 December 2003 recommending the granting of this designation.

Opinions on orphan medicinal products designations are based on the following cumulative 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 5 in 10,000 people in the European Union) 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.

EU/3/03/185: Public summary of positive opinion for orphan designation of tacrolimus hydrate for the treatment of vernal keratoconjunctivitis

AdoptedReference Number: EMEA/COMP/1610/03 Rev. 2

Key facts

• Active substance:
  Tacrolimus Hydrate
• Intended use:
  Treatment of vernal keratoconjunctivitis
• Orphan designation status:
  Withdrawn
• EU designation number:
  EU/3/03/185
• Date of designation:
  14/01/2004
• Sponsor:

  Sucampo Pharma Europe Ltd.; 78 Cannon Street; London EC4 N6NQ; United Kingdom; Telephone: [phone]; Telefax: (44-20) 76 18 86 61; E-mail: [e-mail]