Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in December 2017 on request of the sponsor.
On 15 January 2015, orphan designation (EU/3/14/1413) was granted by the European Commission to Regulatory Resources Group Ltd, United Kingdom, for allogeneic, umbilical cord blood-derived, ex vivo-expanded, haematopoietic CD133+ cells / allogeneic, umbilical cord blood-derived, non-expanded, haematopoietic CD133- cells for the treatment of acute myeloid leukaemia.
Acute myeloid leukaemia (AML) is a cancer of the white blood cells (cells that fight against infections). In patients with AML, the bone marrow (the spongy tissue inside the large bones, where blood cells are produced) produces large numbers of abnormal, immature white blood cells. These abnormal cells quickly build up in large numbers in the bone marrow and are found in the blood.
AML is a long-term debilitating and life-threatening disease because these abnormal immature cells take the place of the normal blood cells, causing bleeding episodes and blood clots, and reducing the patient's ability to fight infections.
At the time of designation, AML affected approximately 1 in 10,000 people in the European Union (EU). This was equivalent to a total of around 51,000 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).
Treatment for AML is complex and depends on a number of factors including the extent of the disease, whether it has been treated before, and the patient's age, symptoms and general state of health. At the time of designation, the main treatments for AML were chemotherapy (medicines to treat cancer) and haematopoietic (blood) stem-cell transplantation (HSCT, a complex procedure where the patient receives stem cells from a matched donor to help restore the bone marrow).
The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with AML because preliminary studies suggest that it could enable more patients to receive HSCT. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
The blood present in the umbilical cord that connects the baby to the placenta (cord blood) is rich in immature stem cells that can be used for HSCT, and which have a lower risk than adult stem cells of being rejected by the donor or of attacking the donor's own cells (graft-versus-host disease). In addition, cord blood can be collected without the need for a surgical procedure. However, because of the small volumes of cord blood, the overall numbers of stem cells collected this way are generally too low to be useful in adults.
The medicine consists of cord blood from which some of the stem cells (called CD133+ cells) have been extracted and grown in the laboratory to increase their numbers, before adding them back to the blood. The enriched cord blood with its increased number of stem cells can then be used to restore normal bone marrow in patients with AML.
The effects of this 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 AML were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for AML. Orphan designation of the medicine had been granted in the United States for this condition.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 11 December 2014 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.
Regulatory Resources Group Ltd
Innovation House
Albany Park
Camberley
Surrey
GU16 7PL
United Kingdom
Tel. +44 (0)1276 671166
Fax +44 (0)1276 670960
E-mail: info@rrgconsultancy.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:
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: