On 20 March 2017, orphan designation (EU/3/17/1852) was granted by the European Commission to Regulatory Resources Group Ltd, United Kingdom, for allogeneic ex-vivo-expanded umbilical cord blood-derived haematopoietic CD34+ progenitor cells and allogeneic non-expanded umbilical cord blood-derived haematopoietic mature myeloid and lymphoid cells (also known as NiCord) for treatment in haematopoietic stem cell transplantation.
The sponsorship was transferred to Voisin Consulting S.A.R.L., France, in January 2019.
In October 2021, Voisin Consulting S.A.R.L. changed name to Voisin Consulting Life Sciences.
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).
At the time of designation, approximately 1 in 10,000 people in the European Union (EU) receive HSCT per year. This was equivalent to a total of around 52,000 people per year*, 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 515,700,000 (Eurostat 2017).
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 because early studies indicate that it can improve success of the transplants. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
The blood present in the umbilical cord that connects the baby to the placenta (cord blood) is rich in immature stem cells that can be used for HSCT, and which have a lower risk than adult stem cells of being rejected by the donor or of graft-versus-host disease. In addition, cord blood can be collected without the need for a surgical procedure. However, because of the small volumes of cord blood, the overall numbers of stem cells collected this way are generally too low to be useful in adults.
The medicine consists of cord blood from which some of the stem cells (called CD34+ cells) have been extracted and grown in the laboratory to increase their numbers, before adding them back to the blood. The enriched cord blood with its increased number of stem cells can then be used to restore normal bone marrow in patients undergoing HSCT.
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 receiving HSCT were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for treatment in HSCT. Orphan designation of the medicine had been granted in the EU for acute myeloid leukaemia, and in the United States for acute myeloid leukaemia, acute lymphoblastic leukaemia, Hodgkin's lymphoma, myelodysplastic syndrome and chronic myeloid leukaemia.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 16 February 2017 recommending the granting of this designation.
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.
Voisin Consulting Life Sciences
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
EMA publishes information on orphan medicinal product designation adopted by the Committee for Orphan Medicinal Products (COMP) on the IRIS online platform:
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.
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: