EU/3/10/763: Orphan designation for the treatment of non-infectious uveitis affecting the posterior segment of the eye

Uveitis is inflammation of the uvea, the middle layer of the eye. The inflammation can be localised only to the back of one or both eyes, and may cause discomfort, pain, and blurring of the vision. Non-infectious uveitis is usually caused by the body's immune system (the body's natural defences) attacking normal tissue and not by an infection.

Non-infectious uveitis affecting the posterior segment of the eye is a long-term debilitating disease because it may lead to partial or complete loss of vision.

At the time of designation, non-infectious uveitis affecting the posterior segment of the eye affected between 0.3 and 1 in 10,000 people in the European Union (EU)*. This is equivalent to a total of between 15,000 and 51,000 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: For the purpose of the designation, the number of patients affected by the condition is estimated and assessed on the basis of data from the European Union (EU 27), Norway, Iceland and Liechtenstein. This represents a population of 506,500,000 (Eurostat 2010).

At the time of designation, several medicines were authorised in Member States of the EU for the treatment of non-infectious uveitis. Most treatments such as corticosteroids aimed to reduce the inflammation by lowering the activity of the immune system. Dexamethasone eye drops and oral solution were authorised for non-infectious uveitis and posterior uveitis, respectively.

The sponsor has provided sufficient information to show that dexamethasone (intravitreal implant) might be of significant benefit for patients with non-infectious uveitis affecting the posterior segment of the eye because results from clinical studies indicate that it might improve the outcome of patients with this condition. In addition, this medicine might contribute to the care of patients because of the way it is expected to carry the dexamethasone to the back of the eye. These assumptions will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

Dexamethasone belongs to a group of anti-inflammatory medicines known as corticosteroids. It helps reduce inflammation by attaching to receptors of immune cells and moderating the activity of the immune system.

Dexamethasone (intravitreal implant) is an implant to be injected into the eye. The implant is made of a biodegradable material which dissolves over several months while slowly releasing dexamethasone inside the eye.

The effects of dexamethasone (intravitreal implant) have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials with dexamethasone (intravitreal implant) in patients with non-infectious uveitis affecting the posterior segment of the eye were ongoing.

At the time of submission, dexamethasone (intravitreal implant) was authorised in the United States of America for macular oedema (swelling of the macula, the central part of the retina).

At the time of submission, dexamethasone (intravitreal implant) was not authorised anywhere in the EU for non-infectious uveitis affecting the posterior segment of the eye. Orphan designation of this medicine had been granted in the United States of America for non-infectious ocular inflammation of the posterior segment in patients with intermediate, posterior and panuveitis.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 6 May 2010 recommending the granting of this designation.

• the seriousness of the condition; t
• he existence of alternative methods of diagnosis, prevention or treatment;
• either the rarity of the condition (affecting not more than 5 in 10,000 people in the EU) 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.