EU/3/14/1388 - orphan designation for prevention of graft-versus-host disease

allogeneic bone marrow derived mesenchymal cells expanded ex vivo in synthetic media

Overview

On 16 December 2014, orphan designation (EU/3/14/1388) 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 prevention of graft-versus-host disease.

What is graft-versus-host disease?

Graft-versus-host disease (GvHD) is a complication that can affect patients who have had allogeneic haematopoietic (blood) stem-cell transplantation. This is a complex procedure used to treat diseases of the blood such as leukaemia (a cancer 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 at risk of the condition?

At the time of designation, the number of people at risk of GvHD was less than 0.4 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 20,000 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).

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*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 28), Norway, Iceland and Liechtenstein. This represents a population of 511,100,000 (Eurostat 2014).

What methods of prevention are available?

At the time of designation, several medicines were authorised in the European Union (EU) for the prevention of GvHD, such as cyclosporine and antilymphocyte immunoglobulins (ATG). 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 this medicine might be of significant benefit for patients at risk of GvHD because early studies showed that it reduced the frequency of GvHD in patients receiving haematopoietic stem-cell 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?

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 matched healthy donor and grown in a laboratory. Mesenchymal cells are able to regulate the activity of 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 the immune cells' ability to attack the patient's organs, thereby preventing 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 for the prevention of GvHD were planned.

At the time of submission, the medicine was not authorised anywhere in the EU for prevention of GvHD 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 13 November 2014 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.

EU/3/14/1388: Public summary of positive opinion for orphan designation: Allogeneic bone marrow derived mesenchymal cells expanded ex vivo in synthetic media for the prevention of graft-versus-host disease

Adopted Reference Number: EMA/COMP/732370/2014

Key facts

• Active substance:
  allogeneic bone marrow derived mesenchymal cells expanded ex vivo in synthetic media
• Intended use:
  Prevention of graft-versus-host disease
• Orphan designation status:
  Positive
• EU designation number:
  EU/3/14/1388
• Date of designation:
  16/12/2014
• Sponsor:

  Cell2B Advanced Therapeutics, SA; Biocant Park; Núcleo 4, Lote 4A; 3060-197, Cantanhede; Portugal; Tel. [phone]; E-mail: [e-mail]

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.