EMA grants orphan drug status to Stemline’s SL-401 for AML treatment

The European Medicines Agency (EMA) has granted Orphan Drug designation to Stemline Therapeutics’ SL-401 for the treatment of acute myeloid leukaemia (AML). SL-401 previously received Orphan Drug designation from the US Food and Drug Administration (FDA) for the treatment of AML and blastic plasmacytoid dendritic cell neoplasm (BPDCN).

SL-401 is a targeted therapy directed to the interleukin-3 receptor (IL-3R) present on cancer stem cells (CSCs) and tumour bulk of BPDCN, AML, and other haematologic cancers. Stemline is evaluating SL-401 in three trials across 7 different indications. The clinical programs include an ongoing pivotal trial in BPDCN, as well as trials in early and late stage AML and several high-risk myeloproliferative neoplasms.