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.

“We are pleased with the EMA’s decision to grant Orphan Drug designation to SL-401 as it underscores the continued unmet need in AML,” noted Eric K. Rowinsky, M.D., Stemline’s chief medical officer and head of research and development. “Additionally, SL-401’s orphan designation from regulators in both the US and Europe provides Stemline with several potential avenues to accelerate clinical development in AML and other orphan indications.”

Stemline Therapeutics, Inc. is a clinical stage biopharmaceutical company developing novel oncology therapeutics that target cancer stem cells (CSCs) and tumour bulk.