Astellas’ experimental acute myeloid leukaemia drug gilteritinib has been awarded orphan drug designation by the European Commission.

The decision indicates that the regulator believes the drug may be of significant benefit to patients with the rare condition, of which there are around 13,000 new cases diagnosed in Western Europe every year.

Gilteritinib is being developed for adult patients with FLT3 mutation-positive relapsed or refractory AML, which is associated with especially poor outcomes.

The drug, originally discovered through a research collaboration with Kotobuki Pharmaceutical, is currently being investigated in Phase III trials.

“We are grateful to the EMA for acknowledging the unique needs of patients with rare diseases, and for providing a potential path forward for gilteritinib in supporting these patients,” noted Steven Benner, global therapeutic area head, Oncology Development, at Astellas.

The drug also holds orphan status in the US.