EU/3/09/618

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Orphan designation

On 27 February 2009, orphan designation (EU/3/09/618) 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 lymphoblastic 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 lymphoblastic leukaemia?

Acute lymphoblastic leukaemia (ALL) is a cancer of the white blood cells called lymphocytes. In this disease, the lymphocytes multiply too quickly and live for too long, so there are too many of them circulating in the blood. These abnormal lymphocytes are not fully developed and do not work properly. Over a period of time, they replace the normal white cells and red blood cells and platelets in the bone marrow (the spongy tissue inside the large bones in the body).

ALL is the most common type of leukaemia in young children, but the disease also affects adults, especially those aged 65 years and older. Many people with acute leukaemia can be cured. However, despite the available treatments, ALL remains a serious and life-threatening disease in some patients.

What is the estimated number of patients affected by the condition?

At the time of designation, ALL affected approximately 1.1 in 10,000 people in the European Union (EU)*. This is below the threshold for orphan designation, which is 5 in 10,000, and is equivalent to a total of around 56,000 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 ALL 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 treatment of ALL is chemotherapy (medicines used to kill cancer cells) followed by or combined with radiotherapy (using radiation to kill cancer cells). Bone-marrow transplantation is also used. This is 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 ALL 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 ALL, 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 ALL were ongoing.

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

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 lymphoblastic leukaemia
Date of decision26/02/2009
OutcomePositive
Orphan decision numberEU/3/09/618

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.