EU/3/13/1227 - orphan designation for treatment of adenovirus infection in allogeneic haematopoietic stem cell transplant recipients
adenovirus-specific T-cells derived from allogeneic donor leukocytes, expanded ex vivo
OrphanHuman
Overview
On 16 January 2014, orphan designation (EU/3/13/1227) was granted by the European Commission to Cell Medica Ltd., United Kingdom, for adenovirus-specific T-cells derived from allogeneic donor leukocytes, expanded ex vivo, for the treatment of adenovirus infection in allogeneic haematopoietic stem cell transplant recipients.
The sponsorship was transferred to Clinical Network Services (NL) B.V., Netherlands, in February 2019.
Please note that this product was withdrawn from the Union Register of orphan medicinal products in January 2020 on request of the Sponsor.
Adenoviruses are a type of virus that can infect human beings, but in healthy people they rarely produce serious illness. However, in patients undergoing allogeneic haematopoietic stem cell transplants (HSCT), which involves replacement of the blood-forming cells in the bone marrow and blood by cells from a healthy donor, the immune system (the body's natural defence) is suppressed by the treatment. Adenovirus infections can therefore lead to inflammation and damage in the lung, the liver, the kidney and the urinary system, the bowel and the brain or spine.
Infection with adenovirus in patients who are given HSCT can be chronically debilitating and life threatening, in particular due to the spread of severe infection.
At the time of designation, adenovirus infection in allogeneic HSCT recipients affected less than 0.2 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 10,000 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 512,200,000 (Eurostat 2013).
At the time of submission of the application for orphan drug designation, no satisfactory methods were authorised for treatment of adenovirus infection in the European Union.
The medicine consists of T-cells, which are a type of white blood cell that fights infection, taken from a healthy donor. These are grown outside the body in the presence of fragments of a protein found in most kinds of adenovirus. This increases the numbers of T-cells and enables them to later recognise and specifically attack virus-infected cells. When they are given to the patient, these T-cells restore the activity that would normally be supplied by the patient's own immune system but which has been suppressed as part of the transplant.
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 this medicine in allogeneic HSCT recipients with adenovirus infection were ongoing.
At the time of submission, this medicine was not authorised anywhere in the EU for adenovirus infection in allogeneic HSCT recipients 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 12 December 2013 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
- adenovirus-specific T-cells derived from allogeneic donor leukocytes, expanded ex vivo
- Intended use
- Treatment of adenovirus infection in allogeneic haematopoietic stem cell transplant recipients
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/13/1227
- Date of designation
- Sponsor
Scendea (NL) B.V.
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: