EU/3/05/261: Orphan designation for the treatment of 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.

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.

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.

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).

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.

• 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.