EU/3/16/1647 - orphan designation for treatment of non-infectious uveitis
Fluocinolone acetonide
OrphanHuman
Overview
Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in July 2017 on request of the sponsor.
On 28 April 2016, orphan designation (EU/3/16/1647) was granted by the European Commission to Campharm Ltd, United Kingdom, for fluocinolone acetonide 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 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 247,000 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 513,700,000 (Eurostat 2016).
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. These included Ozurdex (dexamethasone) given by injection into the eye. Other immunosuppressant medicines such as ciclosporin were also authorised for use in non-infectious uveitis.
The sponsor has provided sufficient information to show that fluocinolone acetonide might be of significant benefit for patients with non-infectious uveitis because the medicine is formulated to provide a prolonged effect over 3 years after injection into the eye, which is much longer than existing treatments. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
Fluocinolone 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 blocking the production of substances that are involved in swelling and inflammation. This medicine is contained in a tiny implant that is injected into the inflamed part of the eye using a special needle. Following injection, the implant releases the corticosteroid over a 3-year period. This helps to ensure that fluocinolone acetonide is delivered long-term to the part of the eye where the inflammation occurs in non-infectious uveitis, and helps avoid transfer to other parts of the body where the medicine is not needed, thus reducing side effects.
The effects of fluocinolone acetonide have been evaluated in experimental models.
At the time of submission of the application for orphan designation, clinical trials with fluocinolone acetonide in patients with non-infectious uveitis were ongoing.
At the time of submission, fluocinolone acetonide was not authorised anywhere in the EU for non-infectious uveitis or designated as an orphan medicinal product elsewhere for this condition. A similar product was available in some EU member states under the trade name Iluvien for the treatment of diabetic macular oedema.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 23 March 2016 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.
Key facts
- Active substance
- Fluocinolone acetonide
- Intended use
- Treatment of non-infectious uveitis
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/16/1647
- Date of designation
- Sponsor
Campharm Ltd
11 Queens Road
Brentwood
Essex CM14 4HE
United Kingdom
Tel. +44 (0)20 3239 6867
E-mail: contact@campharm.eu
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: