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Orphan designation

On 22 May 2012, orphan designation (EU/3/12/969) was granted by the European Commission to DCPrime BV, the Netherlands, for allogeneic human dendritic cells derived from a CD34+ progenitor cell line for the treatment of acute myeloid leukaemia.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

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 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 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 less than 2 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 102,000 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. At the time of designation, this represented a population of 509,000,000 (Eurostat 2012).

What treatments are available?

Treatment of 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. Haematopoietic stem cells are cells in the bone marrow that can develop into different types of blood cells. 

The sponsor has provided sufficient information to show that allogeneic human dendritic cells derived from a CD34+ progenitor cell line might be of significant benefit for patients with AML because it works in a different way to existing medicines which may allow it to be used in combination with other treatments. 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 a type of white blood cell called ‘dendritic cells’ which are produced from immature bone marrow cells (called CD34+ cells) from a donor with AML. These dendritic cells have on their surface specific molecules that are found in large amounts on cancer cells but are not found on normal cells. When the dendritic cells are injected into the patient, the immune system is expected to recognise them as ‘foreign’. This is expected to stimulate an immune response against the AML cells in the body, resulting in the immune system killing the AML cells.

What is the stage of development of this medicine?

At the time of submission of the application for orphan designation, the effects of this medicine had not been evaluated in experimental models.

At the time of submission, clinical trials with the medicine in patients with AML were 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 11 January 2012 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.

Key facts

Product details for <p>Allogeneic human dendritic cells derived from a CD34+ progenitor cell line</p>
Active substanceAllogeneic human dendritic cells derived from a CD34+ progenitor cell line
Medicine Name
Disease/conditionTreatment of acute myeloid leukaemia
Date of decision22/05/2012
Orphan decision numberEU/3/12/969

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

DCPrime BV
Galileiweg 8
2333 BD Leiden
The Netherlands
Tel. +31 20 598 5601
Fax +31 20 598 9904

Patients' organisations

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.