On 12 January 2017, orphan designation (EU/3/16/1817) was granted by the European Commission to Coté Orphan Consulting UK Limited, United Kingdom, for human donor haematopoietic stem and progenitor cells that have been treated ex vivo with the protein transduction domain of the HIV-1 transactivation protein fused to MYC transcription factor (also known as TBX-1400) for treatment in haematopoietic stem cell transplantation.
The sponsorship was transferred to IQVIA RDS Ireland Limited, Ireland, in May 2018.
Haematopoietic stem cell transplantation 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) 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.
Haematopoietic stem cell transplantation 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 0.5 in 10,000 people in the European Union (EU) have haematopoietic stem cell transplantation per year. This was equivalent to a total of around 26,000 people per year*, and is below the ceiling for orphan designation. 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 EU for patients undergoing haematopoietic stem cell transplants. 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 haematopoietic stem cell transplantation because experimental studies showed that the medicine may increase the success of transplantation. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
The medicine is made of donated stem cells and immature blood cells ('haematopoietic stem and progenitor cells') that are to be used in haematopoietic stem cell transplantation. These cells are treated with a specially engineered protein that is made by joining two smaller proteins. One part of the engineered protein enables it to easily enter the cells. Once in the cells, the other part, called MYC protein, transiently stimulates the genes that control the survival and multiplication of stem cells. The medicine when transplanted into a patient is thus expected to increase the production of blood cells and improve success of the transplant.
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 with the medicine in patients receiving haematopoietic stem cell transplantation had been started.
At the time of submission, the medicine was not authorised anywhere in the EU for haematopoietic stem cell transplantation. Orphan designation of the medicine had been granted in the United States for the enhancement of cell engraftment in patients receiving haematopoietic stem cell transplantation.
In accordance with Regulation (EC) No 141/2000 of 16 December 1999, the COMP adopted a positive opinion on 8 December 2016 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.
IQVIA RDS Ireland Limited
Estuary House
East Point Business Park
Fairview
Dublin 3
Ireland
Tel. +353 1819 5100
E-mail: cliona.oleary@iqvia.com
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:
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.
The list of medicines that have received an orphan designation in the EU is available on the European Commission's website: