Astellas’ gilteritinib gets orphan designation in Europe for acute myeloid leukaemia

The decision follows a positive recommendation for Orphan Designation from the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP). In Europe, an Orphan Designation is granted to a medicine that may be of significant benefit to patients with a rare condition, affecting no more than five in 10,000 people.

The announcement follows the recent Orphan Drug Designation in the United States granted by the US Food and Drug Administration (FDA) to gilteritinib on July 13, 2017.

Astellas senior vice president and oncology development global therapeutic area head Steven Benner said: “Around 13,000 people will be diagnosed with AML in Western Europe and, while AML patients constitute a small proportion of the overall population, they are faced with a life-threatening condition.

“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.”

AML is a cancer that impacts the blood and bone marrow, and its incidence increases with age.3 In Western Europe, there are around 13,000 new cases of AML every year.4 In Japan, approximately 4,500 patients are diagnosed with AML each year.

The American Cancer Society estimates that in 2017 approximately 21,000 new patients will be diagnosed with AML in the United States and about 10,000 cases will result in death.