EU/3/21/2504: Orphan designation for the treatment in haematopoietic stem cell transplantation

People who undergo haematopoietic stem cell transplantation may get a condition called GvHD, where transplanted white blood cells recognise the patient as 'foreign' and enter and attack organs such as gut, liver and skin. Movement of these white blood cells into the organs is triggered by a substance called sphingosine-1-phosphate, which attaches to so-called S1P1 receptors on the cells. This medicine acts by preventing activation of S1P1 receptors and so keeping most of the donor's white blood cells in the immune tissue where they cannot attack the cells of the body.

At the time of submission of the application for orphan designation:

• The effects of the medicine had been evaluated in experimental models.

• Clinical trials with the medicine in patients having haematopoietic stem cell transplantation were ongoing.

More information on how potential new medicines are tested during their development is available on Authorisation of medicines.

Medicines intended for rare diseases can be granted an orphan designation during their development.

The orphan designation allows the developer to benefit from:

• scientific and regulatory support to advance their medicine to the stage where they can request marketing authorisation;

• market exclusivity once the medicine is on the market.

To qualify for orphan designation, a medicine must meet a number of criteria:

• it must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;

• the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development;

• there are no satisfactory alternative methods for the diagnosis, prevention or treatment of the condition or the medicine is of significant benefit to those affected by the condition.

EMA's Committee for Orphan Medicinal Products (COMP) is responsible for issuing opinions on applications for orphan designations.

The Agency sends the COMP opinion to the European Commission, which is responsible for granting the orphan designation. The full list of orphan designations is available in the Community register of orphan medicinal products for human use.

For more information, see:

• Orphan designation: Overview

• Rare diseases, orphan medicines: Getting the facts straight