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Orphan designation

On 7 June 2013, orphan designation (EU/3/13/1129) was granted by the European Commission to Cell2B Advanced Therapeutics, SA, Portugal, for allogeneic bone-marrow-derived mesenchymal cells expanded ex vivo in synthetic media for the treatment of graft-versus-host disease.

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

What is graft-versus-host disease?

Graft-versus-host disease (GvHD) is a complication that can affect patients who have received allogeneic haematopoietic (blood) stem-cell transplantation. This is a complex procedure used to treat diseases such as leukaemia or multiple myeloma (cancers of the white blood cells), whereby a patient receives stem cells from a matched donor to help restore the bone marrow, which produces new blood cells.

In GvHD, the transplanted cells recognise the patient as ‘foreign’ and attack the patient’s organs, such as the stomach, gut, skin and liver, leading to organ damage. GvHD may happen shortly after transplantation or later on, in which case a wider range of organs can be involved. GvHD is a serious and life-threatening disease with a high mortality rate.

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

At the time of designation, GvHD affected not more than 0.6 in 10,000 people in the European Union (EU). This was equivalent to a total of not more than 30,000 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 509,000,000 (Eurostat 2013).

What treatments are available?

At the time of designation, several medicines were authorised in the European Union (EU) for GvHD, such as ciclosporin, antilymphocyte immunoglobulins (ATG) and corticosteroids. Treatment aimed to reduce the activity of immune cells involved in GvHD, thereby reducing their ability to attack the patient’s organs.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with GvHD because early studies indicate that it might improve the treatment particularly of patients who do not respond to current 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 an advanced medicinal product that belongs to the group called ‘somatic-cell-therapy products’. These are medicines that contain cells or tissues that have been manipulated to change their biological characteristics so that they can be used to cure, diagnose or prevent a disease.

The medicine is made of mesenchymal cells extracted from the bone marrow of a donor and grown in a laboratory. Mesenchymal cells are able to regulate the immune system. Once injected into the patient’s blood, the mesenchymal cells are expected to reduce the activity of the immune cells involved in GvHD, reducing their ability to attack the patient’s organs, thereby relieving the symptoms of the disease.

What is the stage of development of this medicine?

The effects of the medicine have been evaluated in experimental models.

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

At the time of submission, this medicine was not authorised anywhere in the EU for this condition 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 17 April 2013 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 bone-marrow-derived mesenchymal cells expanded ex vivo in synthetic media</p>
Active substanceallogeneic bone-marrow-derived mesenchymal cells expanded ex vivo in synthetic media
Medicine Name
Disease/conditionTreatment of graft-versus-host disease
Date of decision07/06/2013
Orphan decision numberEU/3/13/1129

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:

Cell2B Advanced Therapeutics SA
Biocant Park
Parque Tecnológico de Cantanhede
Nucleo 4, Lote 4A
3060-197 Cantanhede
Tel. +35 121 121 1157

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.