At the time of designation, several medicines were authorised in the EU for the treatment of myelodysplastic syndromes including azacitidine, lenalidomide and imatinib. The choice of treatment depended on a number of factors, including the type and the extent of the disease, whether it had been treated before, and the patient's age, symptoms and general state of health. The main treatments included medicines that stimulate production of blood cells, chemotherapy (medicines to treat cancer), blood transfusions and stem cell transplantation. Stem cell transplantation is a procedure where the patient's bone marrow is cleared of cells and replaced with stem cells from a donor to form new bone marrow that produces healthy blood cells.

The sponsor has provided sufficient information to show that the medicine might be of significant benefit for patients with myelodysplastic syndromes, because early data showed that some patients did not need blood transfusions or needed fewer transfusions after treatment with the medicine. This assumption will need to be confirmed at the time of marketing authorisation, in order to maintain the orphan status.