EU/3/15/1490: 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 5 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 256,000 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 such as ciclosporin were also authorised for use in non-infectious uveitis.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with non-infectious uveitis because it is given directly into the part of the eye affected by non-infectious uveitis, and early studies have shown that patients experienced fewer side effects compared with authorised corticosteroid treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

Triamcinolone acetonide is a corticosteroid that reduces inflammation and suppresses the body's immune system. It is authorised in various formulations for many inflammatory conditions. It works by entering cells and blocking the production of vascular endothelial growth factor (VEGF) and prostaglandins, substances that are involved in swelling and inflammation. The medicine is expected to be injected into the suprachoroidal space (the portion of the eye below the white part). This will help to ensure that adequate amounts of triamcinolone acetonide are delivered to the part of the eye where the swelling in non-infectious uveitis occurs, and to avoid delivery to other parts of the eye where the medicine is not needed, thus reducing the occurrence of side effects.

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

At the time of submission of the application for orphan designation, clinical trials with the medicine in patients with non-infectious uveitis were ongoing.

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 16 April 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.