EU/3/07/498

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Orphan designation

On 14 November 2007, orphan designation (EU/3/07/498) was granted by the European Commission to S.I.F.I. Società Industria Farmaceutica Italiana S.p.A., Italy, for polihexanide for the treatment of Acanthamoeba keratitis.

What is Acanthamoeba keratitis?

Acanthamoeba are organisms made up of a single cell, which can cause an eye disease. These organisms live and survive in many different places such as air, soil and water environments. From these sources, they can gain access to contact lens solutions and contact lenses. Tap water used to clean lenses is a common source of such an infection. The Acanthamoeba may be on the lens or in the cleaning solutions. If lenses are left in the eyes for long periods of time, these amoebae can multiply and cause damage to the eye (such as rips and ulcers).

Acanthamoeba keratitis is a chronically debilitating condition and may result in loss of sight in the infected eye.

What are the methods of treatment available?

No satisfactory methods exist that were authorised at the time of application.

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, Acanthamoeba keratitis was considered to affect less than 0.1 in 10,000 persons in the European Union, which, at the time of designation, corresponded to about 5,000 persons in total.

*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?

Polihexanide is a disinfectant that is active against bacteria and Acanthamoeba. It is contained in solutions for the preservation of contact lenses, and other medical or industrial products. There are published studies in scientific journals, in which the efficacy of polihexanide in Acanthamoeba keratitis has been evaluated.

What is the stage of development of this medicinal product?

The effects of polihexanide were evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials in patients with Acanthamoeba keratitis had been initiated.

Polihexanide was not authorised anywhere in the world for Acanthamoeba 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>Polihexanide</p>
Active substancePolihexanide
Medicine Name
Disease/conditionTreatment of acanthamoeba keratitis
Date of decision13/11/2007
OutcomePositive
Orphan decision numberEU/3/07/498

Review of designation

Sponsor’s contact details:

S.I.F.I. Società Industria Farmaceutica Italiana S.p.A.
Via Ercole Patti, 36 Lavinaio – Aci S. Antonio - CT
Italy
Telephone: +39 095 7922111
Telefax: + 39 095 7893451

Patients’ associations contact points:

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

The Eyecare Trust
PO Box 804
Aylesbury
Buckinghamshire
HP20 9DF
United Kingdom
Telephone: +44 845 129 5001
Telefax: +44 845 129 5001
E-mail: info@eyecaretrust.org.uk

Action for Blind People 
14-16 Verney Road 
London SE16 3DZ
United Kingdom 
Telephone: +44 20 76 35 48 00
Telefax: +44 20 76 35 49 00 
E-mail: info@afbp.org