EU/3/05/261

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Orphan designation

On 7 March 2005, orphan designation (EU/3/05/261) was granted by the European Commission to Bausch & Lomb (UK) Ltd, United Kingdom, for fluocinolone acetonide (prolonged-release intravitreal implant) for the treatment of non-infectious uveitis affecting the posterior segment of the eye.
The sponsorship was transferred to Bausch & Lomb Ireland in October 2006.

What is non-infectious uveitis affecting the posterior segment of the eye?

The term uveitis covers clinical symptoms of inflammation of the uvea, the middle layer of the eye - between the sclera and retina. The medicinal product, which is the subject of this designation, is a drug delivery system containing fluocinolone acetonide, which is surgically implanted behind the surface of the eye. The condition is, therefore, limited to uveitis affecting the back of the eye.
There are a number of different causes for uveitis. It may result from an infection or it may be related to an autoimmune disease (with or without involvement of other parts of the body), where a reaction to an individual’s own tissues (in this case in the eye) occurs because the body’s defence system rejects other parts of the body. Trauma to the eye can also lead to uveitis. In many cases the cause may be unknown. This medicinal product would only target the non-infectious posterior uveitis.
Uveitis can affect one eye or both eyes and may cause varying degrees of discomfort or pain, with or without blurring of vision. Non-infectious uveitis affecting the posterior segment of the eye is chronically debilitating as it may lead to structural damage to the tissues in the eye, cataracts and sight loss.

What are the methods of treatment available?

Various medicinal products were available for the treatment of non-infectious uveitis affecting the posterior segment of the eye and were authorised in some Member States in the Community at the time of submission of the application for orphan drug designation.
Fluocinolone acetonide (prolonged-release intravitreal implant) might be of potential significant benefit for the treatment of non-infectious uveitis affecting the posterior segment of the eye. The benefit will have to be confirmed at the time of marketing authorisation. This will be necessary to maintain the orphan status.

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

According to the information provided by the sponsor, non-infectious uveitis affecting the posterior segment of the eye was considered to affect between 13,800 to 46,000 persons in the European Union.

*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 25), Norway, Iceland and Lichtenstein. This represents a population of 459,700,000 (Eurostat 2004). 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?

Fluocinolone acetonide is a corticosteroid and is expected to reduce inflammation and suppress autoimmunity. The product is composed of a drug delivery system that is surgically implanted into the posterior segment of the eye to deliver fluocinolone acetonide over a prolonged length of time (target of 3 years).

What is the stage of development of this medicinal product?

The effects of fluocinolone acetonide (prolonged-release intravitreal implant) were evaluated in experimental models. At the time of submission of the application for orphan designation, clinical trials in patients with non-infectious uveitis affecting the posterior segment of the eye were ongoing.
Fluocinolone acetonide (prolonged-release intravitreal implant) was not marketed anywhere worldwide for treatment of non-infectious uveitis affecting the posterior segment of the eye, at the time of submission. On 31 July 2000, orphan designation of flucinolone was granted in the United States for treatment of uveitis involving the posterior segment of the eye.
According to Regulation (EC) No 141/2000 of 16 December 1999, the Committee for Orphan Medicinal Products (COMP) adopted on 13 January 2005 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>Fluocinolone acetonide (prolonged-release intravitreal implant)</p>
Active substanceFluocinolone acetonide (prolonged-release intravitreal implant)
Medicine Name
Disease/conditionTreatment of non-infectious uveitis affecting the posterior segment of the eye
Date of decision06/03/2005
OutcomePositive
Orphan decision numberEU/3/05/261

Review of designation

Related information

Sponsor’s contact details:

Bausch & Lomb Ireland
IDA Industrial Park
Waterford
Ireland
Telephone: + 353 51 35 501
Telefax: + 353 51 35 56 39
E-mail: waterfordinfo@bausch.com

Patients’ associations contact points:

Uveitis Information Group (UIG) 
South House 
Sweening Vidlin 
Shetland Isles 
ZE2 9QE
United Kingdom
Telephone: +44 18 06 57 73 10
E-mail: info@uveitis.net

Geschäftsstelle DUAG e.V. 
Forsthausstrasse 6 
35792 Löhnberg
Germany
Telephone: + 49 64 71 98 05 7 
Telefax: +49 64 71 98 05 8 
E-Mail: duag-ev@web.de

INFLAM’OEIL
France
No postal address available
Telephone: +33 6 12 83 93 63
E-mail: inflamoeil@yahoo.fr