The Biologics License Application (BLA) for Elzonris (tagraxofusp; Stemline Therapeutics) has been accepted for filing and has been granted Priority Review by the Food and Drug Administration (FDA) for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN). 

Elzonris (SL-401) is a novel targeted therapy directed to CD123, a cell surface receptor expressed on a range of malignancies. The Company announced results from a Phase 2 trial involving 45 patients with BPDCN earlier this year. In the Stage 3 cohort, 54% (7/13) met the primary endpoint of complete response + clinical complete response (95% CI: 25.1, 80.8). In first-line patients, overall response rate was 90% with a 72% rate of complete response + complete clinical response; 45% of first-line patients were bridged to stem cell transplant. 

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“Given both Priority and Breakthrough status, our commercial organization is positioning itself to rapidly launch Elzonris, if approved, to ensure this important new treatment reaches patients as quickly as possible,” said Ivan Bergstein, MD, and Stemline CEO. The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of February 21, 2019 for this indication.

Elzonris is also being evaluated in clinical trials for additional indications including chronic myelomonocytic leukemia and myelofibrosis.

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