On 13 May 2011, orphan designation (EU/3/11/867) was granted by the European Commission to Fate Therapeutics LTD, United Kingdom, for allogeneic umbilical cord blood cells treated ex vivo with 16,16-dimethyl prostaglandin E2 for the treatment of acute myeloid leukaemia.
- What is 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) 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 life-threatening disease because these abnormal immature cells take the place of the normal white blood cells, reducing the patient’s ability to fight infections.
- What is the estimated number of patients affected by the condition?
At the time of designation, AML affected not more than 1.2 in 10,000 people in the European Union (EU)*. This is equivalent to a total of not more than 61,000 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 27), Norway, Iceland and Liechtenstein. This represents a population of 506,300,000 (Eurostat 2011).
- What treatments are available?
The 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 (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 this medicine might be of significant benefit for patients with AML because early studies in experimental models show that it might improve the treatment of patients with this condition. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
- How is this medicine expected to work?
This medicine is made up of ‘stem cells’ from the umbilical cord that have been treated with a chemical called ‘16,16-dimethyl prostaglandin E2’ (dmPGE2). Stem cells are cells that can develop into different types of cell. The umbilical cord stem cells are used to replace the cells in the patient’s bone marrow. Treatment with dmPGE2 is expected to improve the properties of these cells, making them better able to survive, multiply, and settle in the patient’s bone marrow.
- What is the stage of development of this medicine?
The effects of this medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, a clinical trial with the medicine in patients with AML was ongoing.
At the time of submission, this medicine was not authorised anywhere in the EU for AML 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 9 February 2011 recommending the granting of this designation.
- Opinions on orphan medicinal product designations are based on the following three criteria
- 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.
|Name||Language||First published||Last updated|
|EU/3/11/867: Public summary of opinion on orphan designation: Allogeneic umbilical cord blood cells treated ex vivo with 16,16-dimethyl prostaglandin E2 for the treatment of acute myeloid leukaemia||(English only)||30/05/2011|
|Active substance||Allogeneic umbilical cord blood cells treated ex vivo with 16,16-dimethyl prostaglandin E2|
|Disease/condition||Treatment of acute myeloid leukaemia|
|Date of decision||13/05/2011|
|Orphan decision number||EU/3/11/867|
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
Sponsor’s contact details
Fate Therapeutics LTD
41 Chalton St, first floor
London NW1 1JD
Tel. +44 (0)20 7554 2252
Fax +44 (0)20 7554 2201
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.