Stemline Therapeutics Announces SL-401 Receives EU Orphan Drug Designation for Treatment of Acute Myeloid Leukemia (AML)

On September 10, 2015 Stemline Therapeutics, Inc. (Nasdaq:STML) reported that the European Medicines Agency (EMA) has granted Orphan Drug designation to SL-401 for the treatment of acute myeloid leukemia (AML). SL-401 previously received Orphan Drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of AML and blastic plasmacytoid dendritic cell neoplasm (BPDCN) (Press release, Stemline Therapeutics, SEP 10, 2015, View Source [SID:1234507443]).

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SL-401 is a targeted therapy directed to the interleukin-3 receptor (IL-3R) present on cancer stem cells (CSCs) and tumor bulk of BPDCN, AML, and other hematologic 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 U.S. and Europe provides Stemline with several potential avenues to accelerate clinical development in AML and other orphan indications."

About Orphan Drug Designation

Orphan Drug designation by the European Commission provides regulatory and financial incentives for companies to develop and market therapies that treat a life-threatening or chronically debilitating condition affecting no more than five in 10,000 persons in the European Union (EU), and where no satisfactory treatment is available. In addition to a 10-year period of marketing exclusivity in the EU after product approval, orphan drug designation provides incentives for companies seeking protocol assistance from the EMA during the product development phase, and direct access to the centralized authorization procedure.