EU/3/20/2271: Orphan designation for the treatment in haematopoietic stem cell transplantation

Haematopoietic stem cell transplantation (HSCT) is a procedure where the patient's bone marrow is cleared of cells and replaced by cells 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 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 0.7 in 10,000 people received HSCT every year in the European Union (EU). This was equivalent to a total of around 36,000 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).

* 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, Iceland, Liechtenstein, Norway and the United Kingdom. This represents a population of 519,200,000 (Eurostat 2020).

At the time of designation, several medicines were authorised in the EU for patients undergoing HSCT. These included medicines that suppress the immune system, such as ciclosporin and corticosteroids, which 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 haematopoietic stem cell transplantation. Early studies have shown that using this medicine for stem cell transplantation may lead to better outcomes than using other blood stem cells from donors.

This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.

This medicine is made of blood stem cells taken from the umbilical cord after it has been removed from a newborn baby. These cells, which have a low risk of causing graft-versus-host disease, are treated and grown in the laboratory to increase their numbers to useful levels. When given to patients undergoing haematopoietic stem cell transplantation, they are expected to lead to fewer cases of graft-versus-host disease and thereby improve patients’ outcomes.

The effects of this medicine have been evaluated in experimental models.

At the time of submission of the application for orphan designation, clinical trials in patients with treatment in haematopoietic stem cell transplantation were ongoing.

At the time of submission, the medicine was not authorised anywhere in the EU for use in haematopoietic stem cell transplantation. Orphan designation had been granted in the United States for the prevention of graft-versus-host disease.

In accordance with Regulation (EC) No 141/2000, the COMP adopted a positive opinion on 19 March 2020, 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.