EU/3/09/619

  • Email
  • Help

Orphan designation

On 27 February 2009, orphan designation (EU/3/09/619) was granted by the European Commission to Teva Pharma GmbH, Germany, for allogeneic ex-vivo-expanded umbilical-cord blood cells for the treatment of acute myeloid leukaemia.

The sponsorship was transferred to Regulatory Resources Group Ltd, United Kingdom, in July 2013.

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 in the body) produces large numbers of abnormal, immature white blood cells called ‘blasts’. 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 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 below the threshold for orphan designation, which is 5 in 10,000, and is equivalent to a total of less than 101,000 people*. This is based on the information provided by the sponsor and 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 504,800,000 (Eurostat 2009).

What treatments are available?

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. The main treatments of AML are chemotherapy (medicines to treat cancer) and bone-marrow transplantation (a complex procedure where the bone marrow of the patient is destroyed and replaced with bone marrow from a matched donor).

The sponsor has provided sufficient information to show that allogeneic ex-vivo-expanded umbilical-cord blood cells might be of potential significant benefit for the treatment of AML because they may be an alternative to donated bone marrow for use in transplantation. 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?

The umbilical cord is the tube that connects an unborn child to its mother until birth. It contains ‘stem cells’, cells that are usually made in the bone marrow and can develop into different types of cell.

Allogeneic ex-vivo-expanded umbilical-cord blood cells come from umbilical cords donated after birth. Because umbilical-cord blood cells are only found in small quantities, the cells are cultivated using a technique called ex-vivo expansion to increase their number.

When allogeneic ex-vivo-expanded umbilical-cord blood cells are transplanted into patients with AML, the stem cells they contain are expected to ‘home’ to the bone marrow and produce normal white blood cells, which will replace the immature blast cells.

What is the stage of development of this medicine?

The effects of allogeneic ex-vivo-expanded umbilical-cord blood cells have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with AML were ongoing.

At the time of submission, allogeneic ex-vivo-expanded umbilical-cord blood cells had not been authorised anywhere in the world for treatment of AML. Orphan designation of the medicine had been granted in the United States for AML.

In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 7 January 2009 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 Community) 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 marketing authorisation.

Key facts

Product details for <p>Allogeneic ex-vivo-expanded umbilical-cord blood cells</p>
Active substanceAllogeneic ex-vivo-expanded umbilical-cord blood cells
Medicine Name
Disease/conditionTreatment of acute myeloid leukaemia
Date of decision26/02/2009
OutcomePositive
Orphan decision numberEU/3/09/619

Review of designation

Sponsor’s contact details:

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

Patients’ associations contact points:

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.