EU/3/15/1507: Orphan designation for the treatment of non-infectious uveitis

Uveitis is inflammation of the uvea, the middle layer of the eye, just beneath the white part of the eye. The inflammation can affect one or both eyes, and may cause discomfort, pain, and blurring of 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 is a long-term debilitating disease because it may lead to partial or complete loss of vision (blindness).

At the time of designation, non-infectious uveitis affected less than 4.8 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 246,000 people*, and is below the ceiling 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 28), Norway, Iceland and Liechtenstein. This represents a population of 512,900,000 (Eurostat 2015).

At the time of designation, several medicines were authorised in Member States of the EU for the treatment of non-infectious uveitis. The first treatment option was corticosteroids, which were used to reduce the inflammation by lowering the activity of the immune system. Other immunosuppressant agents (that reduce the activity of the immune system) such as ciclosporin were also authorised for use in non-infectious uveitis.

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients with non-infectious uveitis because, since the medicine is expected to be given as an injection into the eye, it is expected to have only a local effect in the eye. Use of this medicine may reduce the need to use corticosteroids and immunosuppressants that have effects on the whole body. These assumptions will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

This medicine is expected to block an enzyme called dihydroorotate dehydrogenase (DHODH), which is involved in the production of substances called 'pyrimidines' inside cells. Pyrimidines are needed for the growth of certain cells of the immune system (called auto-reactive T-cells) which in patients with uveitis attack normal tissue and cause inflammation. By reducing the growth of these cells, the medicine is expected to lead to a reduction in inflammation and damage to the uvea, thus improving the symptoms of the condition.

The effects of the medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, no clinical trials with the medicine in patients with non-infectious uveitis had been started.

At the time of submission, the medicine was not authorised anywhere in the EU for non-infectious uveitis or designated as an orphan medicinal product elsewhere for this condition.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 13 May 2015 recommending the granting of this designation.

• the seriousness of the condition;
• the 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.