EU/3/16/1728 - orphan designation for treatment of graft-versus-host disease
recombinant humanised monoclonal antibody against human complement component C5a
OrphanHuman
Overview
Please note that this product was withdrawn from the Community register of designated orphan medicinal products in September 2017 on request of the sponsor.
On 29 August 2016, orphan designation (EU/3/16/1728) was granted by the European Commission to Alexion Europe SAS, France, for recombinant humanised monoclonal antibody against human complement component C5a for the treatment of graft-versus-host disease.
Graft-versus-host disease is a complication that can affect patients who have had allogeneic haematopoietic (blood) stem-cell transplantation to treat diseases of the blood such as leukaemia (a cancer of the white blood cells). The procedure involves a patient receiving stem cells from a matched donor to help restore the bone marrow, which produces new blood cells.
Graft-versus-host disease occurs when 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 happen 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 less than 1 in 10,000 people in the European Union (EU). This was equivalent to a total of fewer than 51,000 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 513,700,000 (Eurostat 2016).
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 to reduce 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 this medicine might be of significant benefit for patients with graft-versus-host disease because preliminary studies suggest that adding the medicine to standard treatments can produce good responses compared with those reported in the published literature. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
This medicine is a monoclonal antibody (a type of protein) that has been designed to attach to and block a protein of the complement system called C5a. The complement system is a group of proteins in the blood that help the immune system (the body's natural defence system) to fight infections. It is thought that activation of the complement system plays a critical role in the development of graft-versus-host disease.
By blocking C5a, the medicine is expected to reduce inflammatory responses caused by the activation of the complement system, thus reducing the organ damage seen in graft-versus-host disease.
The medicine is made by a method known as 'recombinant DNA technology': it is made by cells into which a gene (DNA) has been introduced that makes them able to produce the 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 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 13 July 2016 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
- recombinant humanised monoclonal antibody against human complement component C5a
- Intended use
- Treatment of graft-versus-host disease
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/16/1728
- Date of designation
- Sponsor
Alexion Europe S.A.S.
Review of designation
Please note that this product was withdrawn from the Community register of designated orphan medicinal products in September 2017 on request of the sponsor.
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: