EU/3/17/1958 - orphan designation for treatment in haematopoietic stem cell transplantation
allogeneic umbilical cord blood CD34+ cells cultured ex vivo with Notch ligand Delta1 (dilanubicel)
OrphanHuman
Overview
This medicine is now known as dilanubicel.
On 17 January 2018, orphan designation (EU/3/17/1958) was granted by the European Commission to Voisin Consulting S.A.R.L, France, for allogeneic umbilical cord blood CD34+ cells cultured ex vivo with Notch ligand Delta1 (also known as NLA101) for treatment in haematopoietic stem cell transplantation.
Please note that this product was withdrawn from the Union Register of orphan medicinal products in March 2021 on request of the Sponsor.
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 receive HSCT every 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 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 the medicine might be of significant benefit for patients undergoing HSCT because early studies show that it can improve success of the transplantation and reduce graft-versus-host disease. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.
The medicine consists of CD34+ cells (a type of stem cells) collected from the umbilical cord blood of several donors. CD34+ cells are very effective at multiplying and developing into different types of blood cells when transplanted into the patient. The CD34+ cells in this medicine are cultivated in a laboratory to increase their number. Cord blood cells are less likely than adult stem cells to cause rejection or graft-versus-host disease. The medicine is used off-the-shelf and is given to patients during a HSCT. The CD34+ cells provide a rapid, but short-term, increase in the levels of different blood cells until healthy blood cells are produced by the new bone marrow. This is expected to improve survival 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 undergoing HSCT were ongoing.
At the time of submission, the medicine was not authorised anywhere in the EU for 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 7 December 2017 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 umbilical cord blood CD34+ cells cultured ex vivo with Notch ligand Delta1 (dilanubicel)
- Intended use
- Treatment in haematopoietic stem cell transplantation
- Orphan designation status
- Withdrawn
- EU designation number
- EU/3/17/1958
- Date of designation
- Sponsor
Voisin Consulting S.A.R.L.
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: