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Orphan designation

On 22 February 2018, orphan designation (EU/3/18/1976) was granted by the European Commission to Universitätsmedizin der Johannes Gutenberg-Universität Mainz, Germany, for allogeneic CD4+ and CD25+ T lymphocytes ex vivo incubated with GP120 for the treatment in haematopoietic stem cell transplantation.

What is haematopoietic stem cell transplantation?

Haematopoietic stem cell transplantation (HSCT) is a procedure where the patient's bone marrow is cleared of cells and replaced by stem cells (cells that can develop into different types of cell) from a donor to form new bone marrow that produces healthy blood cells. It can be used to treat serious diseases of the blood and immune system such as leukaemia.

HSCT can be a debilitating and life-threatening procedure due to the risk of severe infections and developing graft-versus-host disease (when the transplanted cells regard the patient’s body as ‘foreign’ and attack the patient’s organs, leading to organ damage).

What is the estimated number of patients receiving haematopoietic stem cell transplantation?

At the time of designation, approximately 1 in 10,000 people receive HSCT per year in the European Union (EU). This was equivalent to a total of around 52,000 people per year*, and is below the ceiling for orphan designation. This isbased 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 28), Norway, Iceland and Liechtenstein. This represents a population of 517,400,000 (Eurostat 2018).

What treatments are available?

At the time of designation, several medicines were authorised in the EU for patients undergoing HSCT. These included radiation treatment or intensive treatment with cancer medicines such as busulfan to clear the bone marrow of existing cells, medicines to help restore the immune system, such as filgrastim, immunoglobulin replacement therapy and Zalmoxis, and medicines to reduce the risk of infections, such as antiviral and antifungal medicines. Medicines that suppress the immune system, such as ciclosporin and corticosteroids, were used for the treatment of graft-versus-host disease.

The sponsor has provided sufficient information to show that this medicine might be of significant benefit for patients undergoing HSCT. This is because laboratory studies showed that the medicine may reduce graft-versus-host disease. Furthermore, the treatment is expected to be used in a different way to other authorised treatments in HSCT including Zalmoxis. 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 consists of a type of white blood cells called regulatory T cells extracted from a donor. Regulatory T cells suppress the activity of other T cells that are responsible for graft-versus-host disease. The cells are treated with a protein called GP120 before being given to the patient along with or after stem cell transplantation. The GP120 treatment helps activate the regulatory T cells and thus reduces the chance of graft-versus-host disease occurring.

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, no clinical trials had been carried out with the medicine in patients undergoing HSCT.

At the time of submission, the medicine was not authorised anywhere in the EU for use in patients undergoing HSCT 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 18 January 2018 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 CD4+ and CD25+ T lymphocytes ex vivo incubated with GP120</p>
Active substanceAllogeneic CD4+ and CD25+ T lymphocytes ex vivo incubated with GP120
Medicine Name
Disease/conditionTreatment in haematopoietic stem cell transplantation
Date of decision22/02/2018
Orphan decision numberEU/3/18/1976

Review of designation

Sponsor’s contact details

Universitätsmedizin der Johannes Gutenberg-Universität Mainz
Department for Dermatology
Langenbeckstr. 1
55131 Mainz
Tel. +49 173 65 52 473

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.