EU/3/09/664

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Orphan designation

On 8 October 2009, orphan designation (EU/3/09/664) was granted by the European Commission to Teva Pharma GmbH, Germany, for allogeneic ex-vivo-expanded umbilical-cord blood cells for the treatment of myelodysplastic syndromes.

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 are myelodysplastic syndromes?

Myelodysplastic syndromes (MDSs) are a group of disorders in which the red blood cells, white blood cells and platelets produced by the bone marrow (the spongy tissue inside the large bones) do not grow and mature normally. Patients with MDSs can develop several symptoms including tiredness or weakness due to anaemia (low red-blood-cell counts), infections due to low white blood cells, and bruising or abnormal bleeding due to low platelet counts.

MDSs are life-threatening diseases because they can lead to severe anaemia, infections or bleeding, and can result in leukaemia (cancer of the white blood cells).

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

At the time of designation, MDSs affected approximately 2.3 in 10,000 people in the European Union (EU). This is equivalent to a total of around 116,000 people*, and is below the threshold for orphan designation, which is 5 people in 10,000. 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?

At the time of designation, some medicines were authorised in the EU for the treatment of MDSs. The choice of treatment for MDSs depended on a number of factors, including the type and 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 for MDSs included chemotherapy (medicines to treat cancer) and bone-marrow transplantation. This is a complex procedure where the bone marrow of the patient is destroyed and replaced with healthy 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 significant benefit for patients with MDSs because they may be an alternative to donated bone marrow for use in transplantation. Advantages of this medicine may include an easier way of obtaining the cells for use in transplantation, the availability of the treatment, particularly to those patients lacking a matching donor, and a reduced delay in finding a matching donor as the medicine is ready to use. These assumptions 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 MDSs, the stem cells they contain are expected to settle in the bone marrow and produce normal white blood cells, red blood cells and platelets, which are missing in patients with MDSs.

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 MDSs were ongoing.

At the time of submission, allogeneic ex-vivo-expanded umbilical-cord blood cells were not authorised anywhere in the EU for MDSs 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 8 July 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 a 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 myelodysplastic syndromes
Date of decision08/10/2009
OutcomePositive
Orphan decision numberEU/3/09/664

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

Patient 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.