EU/3/16/1627 - orphan designation for treatment of post-transplantation lymphoproliferative disorders
Allogeneic Epstein-Barr virus specific cytotoxic T lymphocytes (Tabelecleucel)
OrphanHuman
Overview
On 21 March 2016, orphan designation (EU/3/16/1627) was granted by the European Commission to Wainwright Associates Ltd, United Kingdom, for allogeneic Epstein-Barr virus specific cytotoxic T lymphocytes for the treatment of post-transplant lymphoproliferative disorder.
In November 2016 sponsor, Wainwright Associates Ltd changed name to PharmaLex UK Services Limited.
The sponsorship was transferred to Atara Biotherapeutics Ireland Limited, Ireland, in February 2017.
The medicinal product has been authorised in the EU as Ebvallo since 16 December 2022.
A post-transplant lymphoproliferative disorder is a blood cancer (lymphoma) that occurs after transplantation. Following a transplant, patients receive medicines that weaken their immune system (the body's natural defences) to prevent rejection of the transplant. However, a weakened immune system also makes patients vulnerable to infection with viruses such as the Epstein-Barr virus. The Epstein-Barr virus infects white blood cells called B cells and after transplantation it may cause changes to the infected blood cells leading to cancer.
Post-transplant lymphoproliferative disorder is a life-threatening condition and is debilitating due to weight loss, fever and organ dysfunction.
At the time of designation, post-transplant lymphoproliferative disorder affected approximately 1.6 in 10,000 people in the European Union (EU). This was equivalent to a total of around 82,000 people*, and is below the ceiling for orphan designation, which is 5 people in 10,000. This isbased 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, no satisfactory method were authorised in the European Union for the treatment of post-transplant lymphoproliferative disorder. Patients with the condition were treated with medicines authorised for non-Hodgkin's lymphoma.
This medicine is made of cells of the immune system called T cells that have been taken from a donor. The T cells are first mixed with B cells from the same donor that have been infected with the Epstein-Barr virus so that the T cells learn to recognise infected B cells as 'foreign'. The T cells are then grown to increase their numbers. When the medicine is then given to the patient, the T cells are expected to attack and kill the patient's own infected B cells, helping to control cancers associated with the virus.
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 post-transplant lymphoproliferative disorder were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for post-transplant lymphoproliferative disorder 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 18 February 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
- Allogeneic Epstein-Barr virus specific cytotoxic T lymphocytes (Tabelecleucel)
- Intended use
- Treatment of post-transplantation lymphoproliferative disorders
- Orphan designation status
- Positive
- EU designation number
- EU/3/16/1627
- Date of designation
- Sponsor
Pierre Fabre Medicament
Les Cauquillous
81500 Lavaur
France
Email: sophie.blanchet@pierre-fabre.com
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
The Committee for Orphan Medicinal Products reviewed the orphan designation of Ebvallo at the time of marketing authorisation, and confirmed that the orphan designation should be maintained.
More information is available in the Ebvallo : EPAR - Orphan Maintenance Assessment Report.
Update history
| Date | Update |
|---|---|
| January 2023 | The sponsorship was transferred from Atara Biotherapeutics Ireland Limited to Pierre Fabre Medicament, France. |
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: