Stemline Therapeutics Announces FDA Accepts ELZONRIS BLA & Grants Priority Review

Stemline Therapeutics, Inc. recently announced US FDA has accepted for filing the company’s Biologics License Application (BLA) for ELZONRIS (tagraxofusp; SL-401) for the treatment of patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN). The FDA also granted Priority Review for the BLA and has set a target action date of February 21, 2019, under the Prescription Drug User Fee Act (PDUFA).

The FDA grants Priority Review to product applications that, if approved, would provide significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. ELZONRIS has also been granted Breakthrough Therapy Designation (BTD) and Orphan Drug Designation (ODD) by the FDA.

Ivan Bergstein, MD, Stemline’s CEO, said “The acceptance of our BLA for filing and grant of Priority Review represent tremendous milestones for Stemline and the BPDCN patient community. We would like to thank the patients and their families who participated in our clinical trials, as well as recognize the tireless work of our investigators and entire Stemline team. 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.”

ELZONRIS (tagraxofusp; SL-401) is a novel targeted investigational therapy directed to CD123, a cell surface receptor expressed on a range of malignancies. ELZONRIS successfully completed a pivotal trial in patients with blastic plasmacytoid dendritic cell neoplasm (BPDCN), and a Biologics License Application (BLA) in this indication has been accepted for filing and been granted Priority Review by the US FDA. ELZONRIS has also been granted Breakthrough Therapy Designation (BTD) and Orphan Drug Designation by the FDA. ELZONRIS is also being evaluated in clinical trials in additional indications, including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and others.

Stemline Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing novel oncology therapeutics. Stemline is developing three clinical stage product candidates, ELZONRIS (tagraxofusp; SL-401), SL-801, and SL-701. ELZONRIS is a targeted therapy directed to the interleukin-3 receptor (CD123) present on a range of malignancies. ELZONRIS has completed a pivotal trial in blastic plasmacytoid dendritic cell neoplasm (BPDCN), for which it was granted breakthrough therapy designation (BTD). The pivotal trial met its primary endpoint, and a Biologics License Application (BLA) has been accepted for filing and granted Priority Review by the FDA. ELZONRIS is also being evaluated in clinical trials in additional indications including chronic myelomonocytic leukemia (CMML), myelofibrosis (MF), and others. SL-801 is a novel oral small molecule reversible inhibitor of XPO1 that is currently in a Phase 1 trial of patients with advanced solid tumors; dose escalation is ongoing. SL-701, an immunotherapeutic, has completed a Phase 2 trial in patients with second-line glioblastoma; data and next steps for the program are being evaluated.