EU/3/14/1405 - orphan designation for treatment of non-infectious uveitis
autologous collagen type II-specific regulatory T cells
OrphanHuman
Overview
Please note that this product was withdrawn from the Union Register of orphan medicinal products in November 2019 on request of the sponsor.
On 16 December 2014, orphan designation (EU/3/14/1405) was granted by the European Commission to TxCell, France, for autologous collagen type II-specific regulatory T cells 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 approximately 4.8 in 10,000 people in the European Union (EU). This was equivalent to a total of around 245,000 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 511,100,000 (Eurostat 2014).
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 this medicine might be of significant benefit for patients with non-infectious uveitis because studies in experimental models showed that the product may have improved efficacy compared with available treatments, and its use may result in a reduction in the use of corticosteroids. These assumptions will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine is an advanced therapy medicine that belongs to the group called 'somatic cell therapy products'. These are medicines that contain cells or tissues that have been manipulated to change their biological characteristics so that they can be used to cure, diagnose or prevent a disease.
To prepare this medicine, certain immune cells called regulatory T cells are extracted from the patient's blood and those regulatory T cells that attach specifically to collagen type II, a protein present only in the eye and the joints, are harvested to increase their numbers. They are then given back to the patient, where they preferentially migrate to the inflamed tissue in the eye and are 'activated' by collagen type II, triggering a cascade of activities that lead to a reduction in inflammation and hence to easing of the patient's symptoms.
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 November 2014 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
- autologous collagen type II-specific regulatory T cells
- Intended use
- Treatment of non-infectious uveitis
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/14/1405
- Date of designation
- Sponsor
TxCell
Allée de la Nertière - Les Cardoulines
06560 Valbonne - Sophia Antipolis
France
Tel. +33 497 21 83 01
Fax +33 493 64 15 80
E-mail: contact@txcell.com
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: