EU/3/18/2044 - orphan designation for treatment of graft-versus-host disease
allogeneic bone marrow derived mesenchymal stromal cells, ex-vivo expanded
OrphanHuman
Overview
On 31 July 2018, orphan designation (EU/3/18/2044) was granted by the European Commission to medac Gesellschaft für klinische Spezialpräparate mbH, Germany, for allogeneic bone marrow derived mesenchymal stromal cells, ex-vivo expanded for the treatment of graft-versus-host disease.
Graft-versus-host disease is a complication that can occur in patients who have had a transplant (graft). In this disease, the transplanted cells recognise the patient’s body as ‘foreign’ and attack the patient’s organs, such as the stomach, gut, skin and liver, leading to organ damage. The disease may occur shortly after transplantation or later on, in which case a wider range of organs can be involved.
Graft-versus-host disease is a serious and life-threatening disease with a high mortality rate.
At the time of designation, graft-versus-host disease affected approximately 0.16 in 10,000 people in the European Union (EU). This was equivalent to a total of around 8,000 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 28), Norway, Iceland and Liechtenstein. This represents a population of 517,400,000 (Eurostat 2018).
At the time of designation, several medicines were authorised in the European Union (EU) for the treatment of graft-versus-host disease, such as ciclosporin and corticosteroids. Treatment aimed at reducing the activity of transplanted cells involved in graft-versus-host disease, 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 graft-versus-host disease because early data showed that the medicine was effective in patients in whom corticosteroids and ciclosporin did not work.
This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
The medicine contains human mesenchymal stromal cells (MSCs) from donors which are then grown in a laboratory to increase their numbers. In the body, MSCs help to regulate the immune system, reducing the activation and growth of cells responsible for attacking foreign bodies. The medicine, which is to be given by injection, is expected to reduce immune activity, decreasing inflammation and damage to the affected organs.
The effects of the medicine have been evaluated in experimental models.
At the time of submission of the application for orphan designation, studies with the medicine in patients with graft-versus-host disease were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for graft-versus-host disease 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 21 June 2018 recommending the granting of this designation.
- 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
- Active substance
- allogeneic bone marrow derived mesenchymal stromal cells, ex-vivo expanded
- Intended use
- Treatment of graft-versus-host disease
- Orphan designation status
- Positive
- EU designation number
- EU/3/18/2044
- Date of designation
- Sponsor
medac Gesellschaft für klinische Spezialpräparate mbH
Theaterstraße 6
D-22880 Wedel
Germany
Tel. + 49 4103 8006 0
Fax + 49 4103 8006 100
E-mail: contact@medac.de
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA list of opinions on orphan medicinal product designation
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
Patients' organisations
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
EU register of orphan medicines
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: