On 17 February 2016, orphan designation (EU/3/16/1619) was granted by the European Commission to Eyevensys SA, France, for DNA plasmid encoding a recombinant fusion protein consisting of the extracellular domain of human TNFα p55 receptor linked to the human IgG1 Fc domain for the treatment of non-infectious uveitis.
In May 2017, Eyevensys SA changed name to Eyevensys SAS.
The sponsor’s address was updated in September 2020.
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 approximately 4.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 236,000 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. 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 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 contains genetic material responsible for making the TNF? p55 receptor. This receptor is normally present on the surface of cells of the immune system and attaches to TNF?, a substance that plays a key role in regulating inflammatory processes in non-infectious uveitis. The medicine is expected to be delivered into cells in the eye, where it produces the TNF? p55 receptor. These new receptors attach to TNF? thereby preventing it from attaching to the natural receptor on immune cells and stopping its effect. This is expected to reduce symptoms of inflammation.
The medicine will be given by electrotransfer, a technique where the medicine is first injected into the eye, after which electric pulses are applied to the eye. This will disrupt the cell walls in the eye and allow the medicine to enter cells where it becomes active.
At the time of submission of the application for orphan designation, the evaluation of the effects of this medicine in experimental models was ongoing.
At the time of submission, 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 non-infectious uveitis.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 21 January 2016 recommending the granting of this designation.
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.
Eyevensys S.A.S
11 Rue Watt
Biopark Bâtiment B
75013 Paris
France
Tel. +33(0)184 791060
E-mail: contact@eyevensys.com
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
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.
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: