EU/3/07/498 - orphan designation for treatment of acanthamoeba keratitis
Polihexanide
OrphanHuman
Overview
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.
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.
No satisfactory methods exist that were authorised at the time of application.
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.
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.
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.
- 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
- Active substance
- Polihexanide
- Intended use
- Treatment of acanthamoeba keratitis
- Orphan designation status
- Positive
- EU designation number
- EU/3/07/498
- Date of designation
- Sponsor
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
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: